Archive for the ‘Gene Therapy Research’ Category

A single star may adorn the Texas state flag but the life sciences ecosystem is comprised of a galaxy of brightly shining companies such as FUJIFILM Diosynth Biotechnologies, Taysha Gene Therapies and Veravas developing innovative new medicines, diagnostics and medical devices.

The growth of the quickly rising Lone Star Bio life sciences hub is fueled by financial support from the state government in the form of the multi-billion dollar Cancer Prevention Research Institute of Texas fund and the drug discovery research coming out of the states universities. Baylor College of Medicine and Texas A&M headline the list of academic powerhouses.

Thats where it all begins. Most research that results in drug discovery generally starts at universities, said Andrew Strong, a partner at Houston-based Hogan Lovells, a legal firm representing biotech clients across Texas and the United States, in an interview with BioSpace.

Barry Burgdorf, also an attorney with Hogan Lovells, echoed Strongs statement. There is a ton of great IP spinning out of the universities, he said. That research is fueling a number of startups across the state, as well as legacy pharma companies that are licensing the developmental programs. That, in turn, is strengthening the ecosystem, Burgdorf noted.

Although the slowing economy is reducing expenditures of venture capital, Burgdorf and Strong agreed that in Texas, there has been no slowdown of new technologies being developed in the universities.

Texas is still very focused on growth [and] recruiting companies, Burgdorf said.

Real estate prices are also attractive to companies hoping to set up shop in Texas, especially when compared to other major U.S. hubs such as Boston and San Francisco.

Lower Cost Real Estate

The per-square-foot cost for space in the Boston area is approximately $95.57, according to Pete Briskman, executive managing director and co-lead for JLLs Mid-Atlantic life sciences practice. In the Bay Area, the per-square-foot cost is $82.41. This compares to $22 per square foot In Houston. Briskman said the difference is critical to companies as it can help them build out space and hire new employees.

He said companies used to have a mantra that the real estate costs were less significant than the science. When it comes to a place like Texas where a company is saving tens of millions of dollars, however, it becomes a real consideration.

Strong agreed with that assessment. He relayed that the CEO of a Boston area biotech told him real estate needs were taking up seven percent of its annual budget. The money can go much farther in Texas, he said.

Strong pointed to the August decision of Cellipont Bioservices to relocate to Texas from San Diego. Cellipont, a cell therapy contract development and manufacturing organization, plans to build a 76,000-square-foot facility in the state.

Strong was the founding chief executive officer of Kalon Biotherapeutics, a startup biotech spun out of the A&M system. He sold the company to FUJIFILM.

Strong pointed to the significant investments FUJIFILM has made. Those expenses are having a positive ripple effect across the region. For every one of the 1,000 FUJIFILM jobs in Texas with a salary of more than $80,000, six additional jobs have been created because of these investments, he said.

FUJIFILMs Ever-Expanding Footprint

Since its 2014 arrival in Texas, FUJIFILM Diosynth Biotechnologies (FDB)s contract manufacturing operations in College Station have rapidly expanded and changed the landscape of the ecosystem across the Brazos Valley region. The Japan-based company has invested hundreds of millions of dollars into its facility, expanding its offerings to bolster the development of gene therapies. The College Station facility has become the largest single-use CDMO production campus in the United States.

In 2019, FUJIFILM established anew Gene Therapy Innovation Center. One year later, the federal government selected the College Station facility to support COVID-19 vaccine candidate manufacturing at its Flexible Biomanufacturing Facility.

In December 2021, FUJIFILM announced another investment to expand its services in Texas and in June, it provided additional finances to expand its continuous processing technologies.

Gerry Farrell, chief operating officer at FDB Texas, told BioSpace thecompany's expansions within Texas have been supported by a strong partnership with state and local governments, as well as the universities.

Tayshas Gene Therapies for Rare and Orphan Diseases

Dallas-based Taysha started the year with disappointing news from its experimental gene therapy for Sandhoff and Tay-Sachs diseases, two forms of GM2 gangliosidosis. A patient treated with TSHA-101, a bicistronic vector, died. However, the patient did not succumb to complications from the gene therapy. They died after contracting a hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection while being treated for COVID-19.

Although the death was related to the infection, the independent review board determined a review of the data was warranted.

In addition to TSHA-101, Taysha is also developing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations. In its quarterly financial report issued in August, the companyhighlighted positive momentum with its gene therapy for giant axonal neuropathy.

Taysha received orphan drug and rare pediatric disease designations from the FDA and orphan drug designation from the European Commission for TSHA-120, an AAV9 gene therapy. Data showed GAN patients treated with TSHA-120 have seen durable improvement and recoverability of sensory nerve amplitude potential (SNAP), a definitive clinical endpoint, the company noted in its announcement.

Taysha is also developing a gene therapy for Rett Syndrome. TSHA-102 is the first-and-only gene therapy in clinical development for Rett. It has also received orphan drug and rare pediatric disease designations from the FDA and has been granted orphan drug designation from the European Commission.

Veravas Antibody Detection Platform

Based in Austin, Veravas launched in 2017. The company has developed the VeraPrep Antibody Detection Platform. The platform uses proprietary magnetic beads to pre-analytically clean samples to remove problematic heterophilic and autoantibody interference, according to the company. The clean sample allows for better capture and measurement of targeted IgA, IgG and IgM immunoglobulins.

During the COVID-19 pandemic, Veravas used its platform to develop an antibody test for SARS-CoV-2.

Baylor Genetics' Pandemic Contributions

A pioneer in genetic testing, Houston-based Baylor Genetics offers a range of diagnostic sequencing and analysis. The company provides a full spectrum of cost-effective, genetic testing it claims leads to clinically relevant solutions.

Baylor offers whole exome and genome sequencing services that provide data for point mutations, insertions and deletions. Oncology testing can find mutation panels through next-generation sequencing.

The company also provides prenatal diagnostics, molecular diagnostics and cytogenetics.

During the height of the COVID-19 pandemic, Baylor launched a combination test for the SARS-CoV-2 virus, as well as influenza A and B.

Asuragen

Also based in Austin, molecular diagnostics company Asuragen, a Bio-Techne brand, provides streamlined solutions for genetics, oncology, controls and companion diagnostic needs.

One of Asuragens products is AmplideX, a genetic test for Fragile X syndrome, whichcauses mild to severe intellectual disability and is associated with autism spectrum disorder.

Beyond Fragile X, Asuragen offers a chronic myeloid leukemia monitoring kit.

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5 Life Science Companies Drive Innovation in Lone Star Bio - BioSpace

Duchenne muscular dystrophy (DMD) was first described by the French neurologist Guillaume Benjamin Amand Duchenne in the 1860s, though it took until 1986 for researchers to identify a particular gene flaw that leads to the condition. The identification of the dystrophin gene by Louis Kunkel and Jerry Louis opened the door for disease-modifying therapies such as exon-skipping, stop codon readthrough, gene therapy, and CRISPR/cas9 mediated gene editing that focus in on dystrophin restoration.

Currently, there are 4 drugs approved in the United States for mutations amenable to skipping of exons 51, 53, and 45, which are applicable to about 30% of patients total with DMD. Each of these were approved through the accelerated approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases. At the recently concluded 2022 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) annual meeting, September 21-24, in Nashville, Tennessee, Emma Ciafaloni, MD, gave the Reiner Lecture to a crowd of a few hundred clinicians, highlighting new treatments for DMD.

In her talk, she summarized the expanding pipeline of agents for DMD, how each differs mechanistically, and whether any are more advantageous than another. Ciafaloni, a professor of neurology and pediatrics at the University of Rochester Medical Center, also discussed how to translate new treatments from trials to clinics, the need to improve clinical trial design and process, and how researchers can build on previous successes. Prior to her presentation, as part of a new NeuroVoices, Ciafaloni provided commentary on several topics regarding the DMD pipeline, including the differences and advantages each approach brings, as well as ways to overcome complexities with conducting clinical trials.

Emma Ciafaloni, MD: The exciting research development in the field of Duchenne muscular dystrophy is extraordinary. Many years after understanding the pathophysiology of Duchennewhich the gene wasnt discovered until the late 1980sall that knowledge is finally paying off and opening a window on therapeutic strategies that have to do with disease-modifying gene editing. There are many different approaches now, some like exon skipping, which are already used in the clinics. Some are different stages of development, such as gene therapy in phase three trials. I would be surprised if we didnt have a gene therapy drug in the clinic in the near future. And then CRISPR, which has not been used yet in humans, but has made major milestones and proof of concept in animal models that are highly promising. These are all strategies that are advancing very rapidly, I think that the field is moving much faster than in the past because of the collaboration between pharma and academia, and the patients and the families. There are many clinical trials in Duchenne, and it's a very exciting time.

Also, there has never been a time before in muscular dystrophies in general, not just Duchenne, where there were so many different, new ideas, as well as old ideas that finally started working in humans. The second part of my talk briefly covered other treatments, ideas and strategies that are not directed to restoration of dystrophin. They're not genetic treatments, but they work more on the downstream pathology of muscle degeneration into Duchenne, like the fibrosis, inflammation, and regeneration. There are some interesting drugs out there, probably a few that are going to be approved soon. We're looking at probably a multifactorial type of treatment, it may be a combination treatment. It's never been a richer time in terms of treatments for Duchenne. Also, it's exciting because some of the lessons learn, for example, with the genetic treatments, are extremely helpful for the larger field of neuromuscular diseases and even neurology. The learning has been fantastic.

With spinal muscular atrophy leading the way, we're moving into more muscle-based diseases [with gene therapy], but the lessons learned are still very valuable. Additionally, we have seen this collaboration between different sponsors, pharmaceuticals, and academias to share the learning, because that's just going to help move things faster and better and in a safer way. That is a positive phenomenon that is unprecedented, and it's helping to accelerate the science in a safe and effective way.

There are still many questions that remain. All these genetic modification approaches have been exon skipping, or gene therapy replacement. They don't replace the full-length dystrophin because it's a very large gene. It's a biologically modified type of dystrophin, so there is no doubt that it will have a profound benefit, but I think that there is plenty of room for improvement. Obviously, gene therapy is not approved yet, so remains to be seen in terms of clinical improvement. But even in the exon skipping, I think that we're going to see much more exciting next generation, exon-skipping that people are currently working on very hard on. The field of science and medicine always evolves. What we have now is only going to be much better down the road in a few years. I have no doubt, and the community of Duchenne is working very hard to make even the drugs that we have now, better.

Sometimes, for the more general neurologist or certainly for the general public, it's important to remember that when we talk about Duchenne muscular dystrophy, or many of our neuromuscular diseases that we discuss here at AANEM, these are also rare diseases. The definition from the FDA for a rare disease is less than 200,000 total patients in the United States. For Duchenne, for example, we're talking about maybe around 12,000 patients. This is not [multiple sclerosis], or Parkinson disease or Alzheimer disease. There are challenges in clinical trial designs that are unique, and they need to be understood. Some of the accelerated approval for some of these drugs is part of that challenge and difference. For example, especially with the genetic approach, some of these genetic approaches like exon skipping, only target a specific mutation in maybe 10% to 13% of patients. Now you're taking a subgroup of an ultra-rare disease that is only 10% of that population. Then you need to run clinical trials that are going to have a chance to prove a difference, and so, you restrict the inclusion criteria to a specific age. Then you're really challenged to find enough patients to do well in a placebo-controlled trial. It's important to keep that in mind that there is plenty of room for improvement in making our rare disease clinical trial design more effective, less time consuming for patients, and improving the approval path.

I also want to say that in Duchenne, the amount of data that has been produced in the past several years in terms of motor endpoints, natural history, the six-minute walk test, the North Star [Ambulatory Assessment], etc. These outcome measure prospective cohorts have been incredibly invaluable. This is just to recognize the incredible amount of work that researchers and families and patients have done in the past several years that is helping the field immensely. We are at a different time, its an exhilarating, exciting time. I think that the community of rare diseases like Duchenne have been incredibly, hard-working in a good, cohesive way to advance the field forward, which is very refreshing.

Transcript edited for clarity. Click here for more NeuroVoices.

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NeuroVoices: Emma Ciafaloni, MD, on the Vast Expansion of Innovative Approaches to Duchenne Muscular Dystrophy - Neurology Live

SHANGHAI, Sept. 29, 2022 /PRNewswire/ -- NeuShen Therapeutics, Inc., a biotechnology company focusing on developing innovative treatments for central nervous system (CNS) disorders with dual platforms of AAV-based gene therapy and small molecule discovery, announced today the closure of ~$20 million Series pre-A financing led by LAPAM, a China based venture capital. NeuShen was founded by a group of industrial executives who have extensive global experiences in central nervous system (CNS) drug development. The new capital will be used to expand the team and catalyze in-house CNS drug discovery in both the US and China.

"The successful completion of this fundraising is a testimony to our team's ability to accomplish CNS drug development and jump starts our discovery engine to build a pipeline with AAV-based gene therapy and small molecule programs," said Joan Shen, M.D., Ph.D., chief executive officer and founder of NeuShen. "CNS disease is an area with huge unmet needs. Our company has had a very clear goal from Day 1, which is to develop novel therapies to relieve the burdens of patients with CNS disorders. In the past few months, we have developed an achievable R&D strategy and built-up a substantial core team with experienced CNS drug hunters. Significant progress has been made in building the internal small molecule pipeline and new AAV gene therapy programs. In addition, multiple collaborations and partnerships have been discussed and established."

CNS disorders are increasingly recognized as major causes of death and disability worldwide while the diagnosis and treatments have largely lagged. Urgent measures are needed to tackle the growing challenges. "Bringing breakthroughs and learnings from other disease targets such as ophthalmology, oncology and hematology, we believe AAV-based gene therapy represents a new opportunity in the treatment of CNS disorders. My colleagues and I at Horae Gene Therapy Center are looking forward to working with Neushen to explore these treatment opportunities. The experiences of NeuShen team in neurosciences will be critical to make this happen," noted Dr. Guangping Gao, Professor, Director, Horae Gene Therapy Center, UMass Chan Medical School. The collaborations between NeuShen and UMass are currently in discussion, which will include multiple projects in CNS gene therapy.

"We are very excited to partner with NeuShen from the beginning. Lapam Capital has a strong commitment to healthcare, and we believe the CNS therapeutic area will attract more investment, considering the huge unmet needs and scientific advancements in the field. We highly value Dr. Joan Shen and her management team for their expertise. Lampam Capital is confident with NeuShen's ability to be a top player in developing innovative therapies for CNS diseases," said Mr. Zhihua Yu, Managing Director of Lapam Capital.

"Dr. Shen has assembled a pre-eminent group of scientists, clinicians and drug developers to build a global biotech developing novel medicines for unmet needs in CNS therapeutic areas. TTM Capital are excited to support Neushen to build its multi-modality pipelines to help patients worldwide," said Ms. Lilly Zhang, founding and managing partner at TTM Capital.

About NeuShen Therapeutics

NeuShen Therapeutics is a biotechnology company focusing on innovative drug research and development to address CNS disorders, applying dual research platforms, including AAV-based gene therapy and small molecule discovery. With operations both in Shanghai, China and Boston, MA, NeuShen has a world-class team and is honored to be advised by an outstanding Board of Directors and Scientific Advisory Board.

https://www.neushen.com/

About Lapam Capital

Headquartered in Beijing, Lapam Capital is a leading healthcare venture capital firm in China. Lapam is currently managing five RMB funds and one USD fund, with more than 10 billion RMB under management. Lapam Capital focuses on investments in early to middle stage fast-growing companies that have innovative pharmaceuticals and medical devices. It has invested in about 60 biopharmaceutical companies and 10 medical device companies to date, including Betta Pharma, RemeGen Co. Ltd., Clover Biopharmaceuticals, Yahong Meditech, Stemirna Therapeutics, Binhui Biotech, ImmuneOnco Biopharmaceuticals, Biostar Pharmaceuticals, Aibo Medical Robot Co. Ltd. and many other companies with great potential. Lapam Capital has a professional investment team with more than 20 years' international and domestic biopharmaceutical industry R&D and management experience and can provide comprehensive value-added supports for the invested companies.

About TTM Capital

TTM Capital is an investment firm that specializes in China and worldwide healthcare industry. We focus on early and growth stage companies including pharmaceutical, biotech and medical technology sub-sectors.

TTM Capital's team consists of experienced global vision investment professionals with extensive industry experience, who work together to achieve superior and consistent returns for the firm's investors. We are committed to accumulating industry experience over time, with the aim to develop an ecosystem of expertise to create transformative healthcare businesses.

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SOURCE NeuShen Therapeutics

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NeuShen Therapeutics Closes Pre-A Financing with ~$20M - BioSpace

Presently, there are no effective therapies for spinal cord damage. Physical therapy can help patients regain some movement, but the outcomes are heavily limited in severe cases due to the inability of spinal neurons to repair organically after injury.

However, in a study published in the journal PLOS Biology, Simone Di Giovanni and his research team at Imperial College London (United Kingdom) have looked to change this roadblock. The team displayed how weekly treatments that contain an epigenetic activator can help the regeneration of motor and sensory neurons in the spinal cord, when administered to mice 12 weeks after a serious injury.

Using a small molecule known asTTK21, theresearchers were able to trigger genetic programming that stimulates axon regeneration in neurons. TTK21 affects gene epigenetics via activating the CBP/p300 family of coactivator proteins.

TTK21 therapy was investigated in micemodels where the specimen had experiencedsevere spinal cord damage. The mice were raised in an enriching environment that allowed them to be physically active, as is recommended for human patients.

The treatment started 12 weeks after the severe spinal cord damage and lasted 10 weeks. Researchers discovered numerous improvements following TTK21 therapy when compared to the control treatment. Increased neuron sprouting in the spinal cord was the most noticeable effect. The researchers additionally found that motor axon retraction above the site of injury stopped and sensory axon development sharply increased. These changes were most likely caused by the observed increase in gene expression associated with regeneration. The next stage will be to further increase these effects to stimulate the regenerated axons to reconnect with the rest of the nervous system, so that the mice regain the capability to may move freely again.

Di Giovanni goes on to emphasize, This work shows that a drug called TTK21 that is administered systemically once/week after a chronic spinal cord injury in animals can promote neuronal regrowth and an increase in synapses that are needed for neuronal transmission. This is important because chronic spinal cord injury is a condition without a cure where neuronal regrowth and repair fail. We are now exploring the combination of this drug with strategies that bridge the spinal cord gap such as biomaterials as possible avenues to improve disability in spinal cord injury patients.

Press release:https://www.eurekalert.org/news-releases/964425

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Epigenetic therapy promotes spinal cord regeneration in mice following injury - RegMedNet

PITTSBURGH A collaborative group of neuroscientists from the University of Pittsburgh School of Medicine and Carnegie Mellon University received a $6.8 million grant from the National Institutes of Health Brain Research Through Advancing Innovative Neurotechologies (BRAIN) Initiative to create an ultra-high resolution molecular atlas of the brain and develop brain cell type-specific strategies for effective and precise gene delivery.

The research will leverage genetic information resolved with single-cell precision to establish a comprehensive database of cell types and neural circuits comprising the brains cognitive and reward systems. In combination with ultra-high-resolution magnetic resonance imaging (MRI), the researchers intend to build brain atlases of marmosets and macaque monkeys and make them available to other neuroscientists across the world, free of charge.

This award enables cross-disciplinary collaboration between experts in neural imaging, gene therapy, machine learning, and molecular biology to advance our understanding of single-cell level organization of the brains essential systems, said project principal investigator Dr. William Stauffer, assistant professor of neurobiology at Pitt. We hope this unmatched degree of precision will eventually pave the way for the development of effective and precise gene editing technologies that might revolutionize treatment of previously fatal diseases, such as Alzheimers or Parkinsons.

The recently launched BioForge Initiative, backed by Pitt Senior Vice Chancellor for the Health Sciences, Dr. Anantha Shekhar, will be used to advance the wide-scale production and commercialization of the gene delivery vectors identified with the grant support.

We are excited that the services of a state-of-the-art biomanufacturing facility will soon be available in Pittsburgh to help make the lofty goal of delivering new and improved medical treatments for brain disorders a reality, said Shekhar. It feels very special to participate in a program that will not only bring life-saving treatments to our patients but also facilitate the dissemination of Pitt-developed technologies to research labs around the world and take a big step toward creating products with economic impact on the region.

The BRAIN Initiative was announced in 2013 to deepen understanding of the inner workings of the human mind and over the years has grown to prioritize the expansion of molecular cell-type profiling and data analysis, enabling genetic and non-genetic access to cell types across multiple species. The multi-year NIH grant was awarded as part of the Armamentarium for Precision Brain Cell Access, a large-scale NIH BRAIN Initiative project.

Delivery technologies for specific brain cell types are revolutionizing experimental neuroscience by allowing researchers to probe the cells and circuits underlying complex behaviors, said Dr. John Ngai, director of the NIH BRAIN Initiative. An expanded toolkit of precision brain cell access tools supported by the first phase of the Armamentarium project could ultimately inform cell- and circuit-specific therapies for human patients, for example, those with epilepsy, neurodevelopmental diseases, or mood disorders.

Projects like the one led by Stauffer, who is interested in defining how different cell types contribute to behavior, as well as investigating cell type-specific disease processes, are essential to the Initiatives mission. Stauffer and his close collaborators, Leah Byrne, Ph.D., assistant professor of ophthalmology at Pitt, and Andreas Pfenning, Ph.D., assistant professor of computational biology at CMU, were awarded a BRAIN Initiative grant in 2018 to begin defining the molecular profiles of different neuron types.

Even a small piece of brain tissue contains dozens of different subtypes of neurons, each performing different functions during different behaviors, said Pfenning, who is a part of CMUs Neuroscience Institute. The ability to target these populations using viruses could accelerate basic research and also pave the way for targeted therapeutics.

Pfennings group will use custom-made machine learning models and evolutionary theory to identify sequences that are most likely to label subpopulations of neurons. His laboratory will also test the ability of those sequences to target specific cell types in the mouse brain.

Further building on the molecular profiling data, scientists at Pitts Brain Institute intend to identify cell type-specific drivers of gene expression in the forebrain and the frontal lobe and develop ready-to-use, specific and efficient gene delivery vectors, including adeno-associated viruses (AAVs). To develop novel AAVs, they will use scAAVengr, the single cell AAV engineering pipeline developed by Byrne. The team will combine scAAVengr-optimized AAV viral shells with newly identified cell type-specific enhancers, and the combination of these elements will generate viral vectors capable of delivering highly efficient and cell type-specific gene therapies. Afonso Silva, Ph.D., professor of neurobiology who holds an endowed chair in translational neuroimaging at Pitt and also a member of the Brain Institute, joins Stauffer, Byrne and Pfenning on the project team. The Silva lab will create an ultra-high resolution MRI atlas of the rhesus monkey brain. That MRI-based atlas will provide the framework for detailing how viral vector expression is controlled in a brain-wide fashion.

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Pittsburgh Project to Pave Way for Technology to Revolutionize Treatment of Fatal Brain Diseases - timesobserver.com

NORTH BETHESDA, Md., Sept. 29, 2022 /PRNewswire/ -- The Foundation for the National Institutes of Health (FNIH) and the National Heart Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH) are launching a new partnership to investigate the syndrome of heart failure with preserved ejection fraction (HFpEF). Developing precision treatment strategies for HFpEF is more critical than ever, as the world's population continues to live longer, resulting in an increase in HFpEF cases. Utilizing cutting edge technologies, including digital measurements and artificial intelligence analytic methods, the Accelerating Medicines Partnership Heart Failure (AMP HF) Program is designed to find novel proteins or genes that could mitigate this disease when altered by therapeutics.

"We know that treatments that target the biological changes that drive disease are often most effective, but the challenge faced by researchers is finding the right targets," said Lawrence A. Tabak, D.D.S., Ph.D., who is performing the duties of the NIH director. "AMP Heart Failure aims to improve the odds of hitting the mark earlier and faster."

Although compelling progress has been made in the treatment of many forms of heart disease, death due to heart failure continues to rise nationally. The AMP HF Program aims to alter the landscape of heart failure treatment, improving the outlook for millions of patients around the world.

"The promise of precision treatments for heart failure is that we will have the opportunity to diagnose individuals much earlier and intervene, changing the course of this disease," said NHLBI Director Dr. Gary H. Gibbons. "The AMP Heart Failure program -- and the high caliber of the partnership at its core -- will help us better understand and treat this common syndrome with the goal of ultimately benefitting millions."

In the United States, heart failure directly contributes to about 45% of all cardiovascular disease deaths.1 HFpEF is a common form of heart failure in which the ejection fractionthe percentage of blood ejected from the left ventricle with each heartbeatis within the normal range. HFpEF is difficult to detect, because the left ventricle appears to be functioning normally, and is often deadly, with a five-year survival rate of just 35-40%. In addition to a high risk for mortality, patients with HFpEF live with declining quality of life and poor capacity to perform tasks of daily living.

The AMP HF Program, a public-private partnership facilitated by the FNIH, will advance our understanding of heart failure with preserved ejection fraction using two complementary and integrated research components: analyzing existing HFpEF datasets, sourced from public and private sector funded studies, and initiating a new clinical trial to confirm retrospective findings in an observational cohort with a goal to develop a framework for new precision treatments.

"HFpEF is clearly a major cause of heart failure hospitalizations and diminished quality of life for older patients. Up until now, developing effective therapeutic strategies to identify and treat HFpEF has eluded us. Through AMP HF, we will harness the valuable perspectives and expertise that collaborations bring to biomedical research, paving the way for a more hopeful outlook," said Dr. Julie Gerberding, Chief Executive Officer at the FNIH.

"Roughly half of all heart failure patients suffer from HFpEF. Understanding what it is, when it happens, and how to treat it remains the single largest unmet need in cardiovascular health.2 The AMP HF Program aims to close this gap in understanding, and ultimately improve the lives of patients everywhere," said Dr. Norman Stockbridge, Director of the Division of Cardiology and Nephrology at the Office of Cardiology, Hematology, Endocrinology and Nephrology at the U.S. Food and Drug Administration (FDA).

AMP Heart Failure is the latest initiative to emerge from the AMP Program, a set of public-private collaborations that coalesce the collective knowledge of the NIH, the U.S. FDA, the biotech and pharmaceutical industry, and patient organizations to speed drug development across different diseases. AMP HF brings together the resources of 8 partner organizations spanning the public and private sectors, with combined commitments totaling over $37 million. The FNIH will provide project management for the effort over the next 5 years.

NIH Institutes and Centers involved include:National Heart, Lung, and Blood Institute

Private partners include:American Society of Echocardiography (ASE)Bayer USCytokinetics, Inc.Ionis Pharmaceuticals, Inc.Novartis AGUltromics

Support also provided by the American Heart Association

For more information about the program, click here. To read what our partners and supporters are saying about the program, click here.

About the Accelerating Medicines Partnership Program: AMP Heart Failure joins other AMP programs expediting discovery around Alzheimer's disease, Parkinson's disease, Schizophrenia, Rheumatoid Arthritis and Lupus, Type II Diabetes, Common Metabolic Diseases, Autoimmune and Immune-Mediated Diseases, and the Bespoke Gene Therapy Consortium, all coordinated by the FNIH since the 2014 launch of the large-scale initiative. The AMP partnerships use cutting-edge scientific approaches to bring new medicines to patients by enhancing validation of novel, clinically relevant therapeutic targets and biomarkers. To learn more about AMP, visithttps://fnih.org/AMP.

About the Foundation for the National Institutes of Health: The Foundation for the National Institutes of Health creates and manages alliances with public and private institutions to support the NIH, the world's premier medical research agency. FNIH works with its partners to accelerate biomedical advances and therapies targeting diseases in the United States and across the globe. The FNIH organizes and administers research projects; supports education and training of new researchers; and holds educational events focused on areas of unmet medical need worldwide. Established by Congress in 1990, the FNIH is a not-for-profit 501(c)(3) charitable organization. For additional information about the FNIH, please visit https://fnih.org.

About the National Institutes of Health (NIH): NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visithttps://www.nih.gov/.

ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.

CONTACT:Katherine Thompson[emailprotected]

SOURCE Foundation For The National Institutes of Health, Inc.

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The FNIH Announces New Research Initiative to Identify More Precise Treatment Strategies for Patients Suffering from Heart Failure - PR Newswire

Healthcare Contract Research Outsourcing Market: Introduction

According to the report, the global healthcare contract research outsourcing market was valued at US$ 38.04 Bn in 2020 and is projected to expand at a CAGR of 6.6% from 2021 to 2028. A contract research organization (CRO) is a company that provides clinical trial management services for the pharmaceutical, biotech, and medical device companies.

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The different types of CRO services are regulatory affairs, site selection & activation, recruitment support, clinical monitoring, data management, trial logistics, pharmacovigilance, biostatistics, medical writing, and project management.

Broad and Expanding Product Pipeline: Key Driver

Rise in demand for new drug and novel therapies for treatment of life-threatening diseases such as cancer & immunological disorders and evolving research & development process have led to an increase in the number of compounds in pipeline in the last decade.

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According to the Congressional Budget Office (CBO), in 2019, there was an increase of 60% of new drugs approved for sale compared to that in 2018.

In the past few years, development of biosimilar products, cellular therapy, and immune-biological has contributed toward the advancing pipeline and need of stringent clinical trials. This drives the global healthcare contract research outsourcing market.

Funding for Emerging Healthcare Companies: Major Driver

Increase in funding for small to mid-sized pharmaceutical, biotechnology, and medical devices companies has induced large companies to opt for CRO services with focus on niche market. Currently, expenditure in the biotechnology sector is the highest due to recent advancements in research on monoclonal antibodies, immunotherapy, gene therapy, and cancer vaccines. Small pharmaceutical companies focus on advancement in product development through CRO services due to rise in demand for innovative products and patent expiration.

The need to improve adoption and value proposition in the market has induced medical devices companies to outsource clinical trial services.

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Clinical Trial Services Segment to Dominate Global Market

The clinical trial services segment is expected to dominate the global market during the forecast period. Optimization of R&D costs and shorter drug development timeline resulting from outsourcing of clinical trial services to CROs are likely to contribute to the segments large market share in the near future. However, the segment is projected to lose market share to regulatory services and clinical data management & biometrics segments by 2028 due to stringent regulatory guidelines.

North America to Lead Healthcare Contract Research Outsourcing Market

In terms of region, the global healthcare contract research outsourcing market has been segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America accounted for a major share of 46.59% of the global market in 2020. The market in the region is estimated to reach US$ 27.19 Bn in 2028, expanding at a CAGR of 5.6% from 2021 to 2028. This can be attributed to the presence of world renowned research experts in CNS, rare disease, oncology, immunology, and stem cells, advanced infrastructure of clinical research sites, and effective government incentive programs.

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Key Players in Global Market

Major players operating in the global healthcare contract research outsourcing market include Syneos Health, PAREXEL International, ICON plc, PRA Health Sciences, Inc., Charles River, Laboratory Corporation of America Holdings (Covance), IQVIA, Medpace, and Pharmaceutical Product Development, LLC.

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Healthcare Contract Research Outsourcing Market to Reach the Value of US$ 63.09 Bn by 2028 - Digital Journal

Only companies that have strong platforms will be able to meet specific timelines, says Bristol-Myers Squibbs Jim Xu.

As COVID-19 transitions into an endemic, delegates at Biotech Week Boston discussed the challenges concerning the upstream process development process. Jianlin (Jim) Xu, scientific director, biologics development at BMS, explained told a packed room that monoclonal antibodies (mAbs) are difficult to produce with complex glycosylation profile, process related impurities, and amino acid oxidation.

Xu also outlined other challenges, which included bioreactor operation scalability, upstream processes lasting over a month for one run from vial thaw to harvest, competitive development of similar mAb products in the industry, and the length of time it takes to create stable monoclonal CHO lines with desirable characteristics.

To solve said challenges, Xu maintained that a strong platform is the number one solution, and that a robust platform can help a firm meet particular timelines. He said that a platform approach has numerous benefits, from decreasing costs, shortening development timelines, and making it possible to have a robust production process from clinical supply through to commercial use.

Xu claimed that in order to develop upstream platform technology, the individual and/or company must be able to demonstrate the benefit and proof-of-concept of an upstream technology in the first instance. Additionally, he told delegates that the technology should be successfully applied to various mAb products, and this will provide a good starting point for future mAb manufacture process development.

In other platform related news at Biotech Week Boston, PerkinElmer launched its Cellaca PLX system, saying that it is the cell analysis solution to streamline cell and gene therapy research and production.

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BMS: Platforms are pivotal to pandemic speed - BioProcess Insider - BioProcess Insider

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INTRODUCTION Over the last few decades, the development landscape of small molecule drugs has been significantly impacted by various biotechnology breakthroughs. Further, with the advent of novel technologies, biologics have made a significant impact in the pharmaceutical domain, delivering ground-breaking treatment for a myriad of disease indications, including immunological, oncological and rare disorders.

New York, Sept. 29, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Biopharmaceutical Contract Manufacturing Market by Type of Service Offered, Type of Biologic Manufactured, Type of Expression System Used, Scale of Operation, Company Size, and Key Geographical Regions : Industry Trends and Global Forecasts, 2022 2035" - https://www.reportlinker.com/p06323412/?utm_source=GNW In this context, it is worth highlighting those 14 biopharmaceutical products (including cell therapies, gene therapies, monoclonal antibodies and recombinant proteins) were approved in the US alone, in 2021. Further, promising results from ongoing clinical research initiatives have encouraged various government and private firms to make significant investments in this domain. For instance, in 2021, a sum of over USD 70 billion was invested in the cell and gene therapy domain. However, manufacturing of biologics is fraught with various challenges. Some of the key concerns of contemporary innovators include rate of attrition of pipeline drugs / therapies, prolonged development timelines, current facility limitations, regulatory and compliance-related issues, and inconsistencies related to quality attributes of the final product. Therefore, therapy developers are actively exploring avenues that enable them to overcome the existing challenges. Amongst other alternatives, outsourcing has emerged as a lucrative option for biologic drug developers.

Currently, a significant number of players engaged in the biopharmaceutical domain prefer to outsource various operations to contract service providers. In fact, currently over 275 companies claim to offer contract manufacturing services for biologic therapeutics, in compliance with the regulatory standards. It is also worth highlighting that biopharmaceutical contract manufacturers are actively trying to consolidate their presence in this field by entering into strategic alliances in order to meet the indubitably rising demand for biologics. For this purpose, substantial expansions, and mergers and acquisitions have been reported in this market, as service providers strive to become one-stop-shops, to cater to the diverse needs of their clientele. With outsourcing being increasingly accepted as a viable and beneficial business model within this field, we anticipate the biopharmaceutical contract manufacturing market to grow at a commendable pace in the coming years.

SCOPE OF THE REPORTThe Biopharmaceutical Contract Manufacturing Market by Type of Service(s) Offered (API, FDF), Type of Biologic Manufactured (Antibodies, Cell Therapies, Vaccines and Other Biologics), Type of Expression System Used (Mammalian, Microbial and Others), Scale of Operation (Preclinical / Clinical and Commercial), Company Size (Small, Mid-sized, and Large and Very Large), and Key Geographical Regions (North America, Europe, Asia-Pacific, Latin America and MENA): Industry Trends and Global Forecasts, 2022 2035 report features an extensive study of the current market landscape and the likely future potential associated with the biopharmaceutical contract manufacturing market, over the next decade.

The study also includes a detailed analysis of key drivers and trends within this evolving market.

Amongst other elements, the report features:

A detailed overview of the overall landscape of companies engaged in offering contract manufacturing services for biologics, including a detailed analysis based on several relevant parameters, such as year of establishment, company size (based on number of employees), location of headquarters, type of service(s) offered (API and FDF manufacturing), type of biologic manufactured (ADCs, antibodies, biosimilars, cell therapies, gene therapies, nucleic acids / oligonucleotides, plasmid DNA / viral vectors, proteins / peptides, vaccines and others), scale of operation (preclinical, clinical and commercial), type of expression system used (mammalian, microbial and others), type of bioreactor used (single use, stainless steel and others) and mode of operation of bioreactor (batch, fed batch and perfusion / continuous).A detailed landscape of the biopharmaceutical manufacturing facilities established across the key geographical regions (North America, Europe, Asia-Pacific and Rest of the World), highlighting the manufacturing hubs for biologics.Elaborate profiles of key industry players based in North America, Europe and Asia-Pacific that offer contract manufacturing services for biologics. Each profile features a brief overview of the company, details related to its biologic-related service portfolio, manufacturing facilities, recent developments, and an informed future outlook.A detailed discussion on the key enablers in this domain, including certain niche product classes, such as antibody drug conjugates (ADCs), bispecific antibodies, cell therapies, gene therapies and viral vectors, which are likely to have a significant impact on the growth of the contract services market.A case study on the growing global biosimilars market, highlighting the associated opportunities for biopharmaceutical CMOs and CDMOs.A case study comparing the key characteristics of small and large molecule drugs, along with details on the various steps involved in their respective manufacturing processes.A detailed discussion on the benefits and challenges associated with in-house manufacturing, featuring a brief overview of the various parameters that a drug / therapy developer may need to take into consideration while deciding whether to manufacture its products in-house or outsource the production operations.A qualitative analysis, highlighting various factors that need to be taken into consideration by biopharmaceutical developers while deciding whether to manufacture their respective products in-house or engage the services of a CMO.A review of the various biopharmaceutical-focused manufacturing initiatives undertaken by top 10 big pharma players (shortlisted on the basis of 2021 revenues), highlighting trends across various parameters, such as number of initiatives, year of initiative, purpose of initiative, type of initiative, scale of operation and type of biologic manufactured.An analysis of the recent collaborations within the biopharmaceutical contract manufacturing industry, based on several relevant parameters, such as year of partnership, type of partnership, type of biologic manufactured, therapeutic area, most active players (in terms of number of deals inked) and regional distribution of partnership activity that have taken place in this domain, during the period 2015-2022.A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of companies, type of acquisition, type of biologic manufactured and key value drivers.A detailed review of expansion initiatives undertaken by biopharmaceutical contract manufacturers, during the period 2016-2022, along with information on several relevant parameters, such as year of expansion, purpose of expansion, type of biologic manufactured and location of expanded facility.An analysis of the recent developments within the biopharmaceutical contract manufacturing industry, highlighting information on the funding investments made during the period 2016-2022, along with information on the technology advancements related to biomanufacturing.An estimate of the overall, installed capacity for the manufacturing of biopharmaceuticals, based on information reported by various industry stakeholders in the public domain, highlighting the distribution of the available capacity, based on size of manufacturer (small, mid-sized and large and very large), scale of operation (preclinical, clinical and commercial), type of expression system used (mammalian, microbial and others) and geography (North America, Europe, Asia-Pacific and Rest of the World).An informed estimate of the annual demand for biologics, taking into account the top 20 biologics, based on various relevant parameters, such as target patient population, dosing frequency and dose strength of the abovementioned products.A company size-wise, detailed analysis of the total cost of ownership for biopharmaceuticals contract manufacturing organizations, during the period 2022-2042.A case study on the virtual business model concept, along with its role in the overall biopharmaceutical industry. It also features a discussion on the advantages and risks / challenges associated with outsourcing operations from virtual service providers.A discussion on affiliated trends, key drivers and challenges, under an elaborate SWOT framework, which are likely to impact the industrys evolution, including a Harvey ball analysis, highlighting the relative effect of each SWOT parameter on the overall biopharmaceutical industry.A survey analysis featuring inputs solicited from various experts who are directly / indirectly involved in providing contract manufacturing services to biopharmaceutical developers.

One of the key objectives of the report was to estimate the existing market size and estimate the future size of biopharmaceutical contract manufacturing market. We have provided informed estimates on the evolution of the market, over the period 2022-2035. Our year-wise projections of the current and future opportunity have further been segmented on the basis of [A] type of service(s) offered (API, FDF), [B] type of biologic manufactured (antibodies, cell therapies, vaccines and other biologics), [C] type of expression system used (mammalian, microbial and others), [D] scale of operation (preclinical / clinical and commercial), [E] company size (small, mid-sized, and large and very large), and [F] key geographical regions (North America, Europe, Asia-Pacific, Latin America and MENA).

In order to account for future uncertainties associated with some of the key parameters and to add robustness to our forecast model, we have provided three market forecast scenarios, portraying the conservative, base and optimistic tracks of the markets evolution.

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

KEY QUESTIONS ANSWEREDWho are the key players engaged in offering contract manufacturing services for biopharmaceuticals?What are the different partnerships and expansion initiatives undertaken by biopharmaceutical contract manufacturers in the recent past?Which regions represent the current key contract manufacturing hubs for biopharmaceuticals?What is the current, installed capacity for contract manufacturing of biopharmaceuticals?What is the current, global demand for biologics? How is the demand for such candidates likely to evolve in the foreseen future?What percentage of the biopharmaceuticals manufacturing operations are presently outsourced?What factors should be taken into consideration while deciding whether the manufacturing operations for biopharmaceuticals should be kept in-house or outsourced?How is the current and future opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 is an executive summary of key insights captured during our research. It offers a high-level view on the current state of biopharmaceutical contract manufacturing market and its likely evolution in the short to mid-term and long-term.

Chapter 3 provides a general introduction to biopharmaceuticals and biopharmaceutical manufacturing processes. The chapter also includes an overview of the various expression systems used for the development of different types of biotherapeutic products. It features a brief overview of contract manufacturing, along with a detailed discussion on the need for outsourcing within the biopharmaceutical industry. Furthermore, it provides information on the challenges faced by players currently engaged in this domain.

Chapter 4 provides a detailed assessment of the current market landscape of companies engaged in offering contract manufacturing services for biologics, including a detailed analysis based on several relevant parameters, such as year of establishment, company size (based on number of employees), location of headquarters, type of service(s) offered (API and FDF manufacturing), type of biologic manufactured (ADCs, antibodies, biosimilars, cell therapies, gene therapies, nucleic acids / oligonucleotides, plasmid DNA / viral vectors, proteins / peptides, vaccines and others), scale of operation (preclinical, clinical and commercial), type of expression system used (mammalian, microbial and others), type of bioreactor used (single use, stainless steel and others) and mode of operation of bioreactor (batch, fed batch and perfusion / continuous).

Chapter 5 provides a detailed landscape of the biopharmaceutical manufacturing facilities established across the key geographical regions (North America, Europe, Asia-Pacific and Rest of the World), highlighting the manufacturing hubs for biologics.

Chapter 6 provides elaborate profiles of key industry players based in North America that offer contract manufacturing services for biologics. Each profile features a brief overview of the company, details related to its biologic-related service portfolio, manufacturing facilities, recent developments, and an informed future outlook.

Chapter 7 provides elaborate profiles of key industry players based in Europe that offer contract manufacturing services for biologics. Each profile features a brief overview of the company, details related to its biologic-related service portfolio, manufacturing facilities, recent developments, and an informed future outlook.

Chapter 8 provides elaborate profiles of key industry players based in Asia-Pacific that offer contract manufacturing services for biologics. Each profile features a brief overview of the company, details related to its biologic-related service portfolio, manufacturing facilities, recent developments, and an informed future outlook.Chapter 9 provides a detailed discussion on the key enablers in this domain, including certain niche product classes, such as antibody drug conjugates (ADCs), bispecific antibodies, cell therapies, gene therapies and viral vectors, which are likely to have a significant impact on the growth of the contract services market.

Chapter 10 presents a case study on the growing global biosimilars market, highlighting the associated opportunities for biopharmaceutical CMOs and CDMOs.

Chapter 11 presents a case study comparing the key characteristics of small and large molecule drugs, along with details on the various steps involved in their respective manufacturing processes.

Chapter 12 provides a detailed discussion on the benefits and challenges associated with in-house manufacturing, featuring a brief overview of the various parameters that a drug / therapy developer may need to take into consideration while deciding whether to manufacture its products in-house or outsource the production operations.Chapter 13 presents a qualitative analysis, highlighting various factors that need to be taken into consideration by biopharmaceutical developers while deciding whether to manufacture their respective products in-house or engage the services of a CMO.

Chapter 14 provides a review of the various biopharmaceutical-focused manufacturing initiatives undertaken by top 10 big pharma players (shortlisted on the basis of 2021 revenues), highlighting trends across various parameters, such as number of initiatives, year of initiative, purpose of initiative, type of initiative, scale of operation and type of biologic manufactured.

Chapter 15 presents an analysis of the recent collaborations within the biopharmaceutical contract manufacturing industry, based on several relevant parameters, such as year of partnership, type of partnership, type of biologic manufactured, therapeutic area, most active players (in terms of number of deals inked) and regional distribution of partnership activity that have taken place in this domain, during the period 2015-2022.

Chapter 16 provides a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2022, based on several relevant parameters, such as year of agreement, type of deal, geographical location of companies, type of acquisition, type of biologic manufactured and key value drivers.

Chapter 17 presents a detailed review of expansion initiatives undertaken by biopharmaceutical contract manufacturers, during the period 2016-2022, along with information on several relevant parameters, such as year of expansion, purpose of expansion, type of biologic manufactured and location of expanded facility.

Chapter 18 presents an analysis of the recent developments within the biopharmaceutical contract manufacturing industry, highlighting information on the funding investments made during the period 2016-2022, along with information on the technology advancements related to biomanufacturing.

Chapter 19 provides an estimate of the overall, installed capacity for the manufacturing of biopharmaceuticals, based on information reported by various industry stakeholders in the public domain, highlighting the distribution of the available capacity, based on size of manufacturer (small, mid-sized, and large and very large), scale of operation (preclinical, clinical and commercial), type of expression system used (mammalian, microbial and others) and geography (North America, Europe, Asia-Pacific and Rest of the World).

Chapter 20 presents an informed estimate of the annual demand for biologics, taking into account the top 20 biologics, based on various relevant parameters, such as target patient population, dosing frequency and dose strength of the abovementioned products.

Chapter 21 presents a company size-wise, detailed analysis of the total cost of ownership for biopharmaceuticals contract manufacturing organizations, during the period 2022-2042.

Chapter 22 presents an insightful market forecast analysis, highlighting the future potential of biopharmaceutical contract manufacturing market till 2035. We have segmented the market on the basis of type of service(s) offered (API, FDF), type of biologic manufactured (antibodies, cell therapies, vaccines and other biologics), type of expression system used (mammalian, microbial and others), scale of operation (preclinical / clinical and commercial) , company size (small, mid-sized, and large and very large), and key geographical regions (North America, Europe, Asia-Pacific, Latin America and MENA).

Chapter 23 presents a case study on the virtual business model concept, along with its role in the overall biopharmaceutical industry. It also features a discussion on the advantages and risks / challenges associated with outsourcing operations from virtual service providers.

Chapter 24 provides a discussion on affiliated trends, key drivers and challenges, under an elaborate SWOT framework, which are likely to impact the industrys evolution, including a Harvey ball analysis, highlighting the relative effect of each SWOT parameter on the overall biopharmaceutical industry.

Chapter 25 features an elaborate discussion on the future opportunities / trends within the biopharmaceutical contract manufacturing market that are likely to influence the growth of this domain over the coming years.

Chapter 26 provides a survey analysis featuring inputs solicited from various experts who are directly / indirectly involved in providing contract manufacturing services to biopharmaceutical developers.

Chapter 27 is a summary of the entire report. It provides the key takeaways and presents our independent opinion of the biopharmaceutical contract manufacturing market, based on the research and analysis described in the previous chapters.

Chapter 28 is a collection of transcripts of interviews conducted with various stakeholders in the industry.

Chapter 29 is an appendix, which provides tabulated data and numbers for all the figures provided in the report.

Chapter 30 is an appendix, which provides the list of companies and organizations mentioned in the report.Read the full report: https://www.reportlinker.com/p06323412/?utm_source=GNW

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Biopharmaceutical Contract Manufacturing Market by Type of Service Offered, Type of Biologic Manufactured, Type of Expression System Used, Scale of...

Introduction

Huntingtons disease (HD) is an autosomal dominant neurodegenerative disorder with an estimated prevalence of up to 9 per 100,000 in the USA, Canada, Oceania, and Western Europe.1,2 HD is caused by a CAG (cytosine, adenine, and guanine) repeat expansion in exon 1 of the Huntingtin (HTT) gene, resulting in the translation of a mutant Huntingtin protein harboring a toxic polyglutamine (polyQ) stretch at its amino (N) terminus. Gene carriers with repeats between 36 and 39 CAG show incomplete penetrance, while repeats of 40 and more triplets lead to fully penetrant disease. The age of onset is inversely correlated with the CAG repeat length, with an average age of onset of 3544 years. HD is characterized by motor, cognitive and psychiatric symptoms and is ultimately fatal, with a median survival of 1518 years after onset. About 510% of HD patients show disease onset before 20 years of age, in which case it is called juvenile HD. Juvenile HD has a different clinical presentation compared to adult onset HD, characterized by symptoms such as severe mental retardation, speech and language delay, as well as more pronounced motor and cerebellar symptoms and overall more rapid disease progression.3

Apart from the inherited CAG length, several genetic modifiers have been identified that are associated with age of onset. Many of these modifiers point towards an important role for somatic instability: the process in which the CAG repeat within cells expands over time. Within the HTT locus, a strong genetic modifier is whether or not a CAA (cytosine, adenine, and adenine) interruption is present at the 3 end of the CAG repeat. Similar to CAG triplets, CAA encodes for glutamine, thus resulting in the same polyQ stretch. Nonetheless, alleles that lack this CAA interruption were found to be more prone to somatic expansion and showed decreased age of onset, while the presence of an additional CAA interruption was found to delay both somatic expansion and age of onset.4,5 Moreover, many of the identified trans-acting genetic modifiers, such as FANCD2 And FANCI Associated Nuclease 1 (FAN1) and MutL Homolog 1 (MLH1), are involved in DNA mismatch repair and influence somatic instability of the CAG repeat.5,6

Although HD was initially thought to be mainly a protein toxic gain-of-function disorder, it is likely that protein loss-of-function also plays a role, as reviewed elsewhere,710 and there is increasing evidence for the involvement of other disease mechanisms, such as repeat-associated non-AUG dependent (RAN) translation and RNA toxic gain-of-function, also reviewed previously.1113 Still, little is known regarding the relative contribution of each of these pathogenic mechanisms to the disease (Figure 1).

Figure 1 Schematic overview of the molecular pathogenesis of HD.

HTT is known to be essential for embryonic development, as demonstrated by the fact that knockout mice are embryonically lethal, and also appears to play a role in later stages of development and life, as reviewed by Kaemmerer and Grondin.10 There is, however, no clear consensus on the level of wild type HTT (wtHTT) that is required for its normal function, as this is likely to depend on many factors, including age and tissue/brain region. wtHTT is involved in many important cellular processes, including endocytosis and vesicular trafficking, cell division, autophagy and transcriptional regulation (reviewed by Saudou and Humbert)9 which may all be impacted by a loss of wtHTT function in HD.

Compelling evidence for the involvement of RNA-mediated toxicity was provided by Sun et al, who found that even in the absence of translation, there was still repeat-length dependent toxicity of 5 HTT mRNA as well as full-length HTT.14 RNA toxic gain-of-function is caused by the interaction between RNA-binding proteins (RBPs), such as Muscleblind like splicing regulator 1 (MBNL1) and Pre-mRNA processing factor 8 (PRPF8), and the secondary structure formed by the expanded CAG repeat in the mRNA, affecting the splicing of a range of transcripts.15,16 This interaction appears to be dependent on the purity of the CAG repeat (ie, the absence of CAA interruptions), as Mbnl1 was found to be recruited to nuclear foci in the novel BAC-CAG mouse model, which has an uninterrupted repeat, but not in the BACHD model, which harbors an interrupted repeat.17

Finally, the presence of the expanded CAG repeat has also been shown to induce repeat-associated non-AUG dependent (RAN) translation, which leads to the production of homopolymers other than polyQ that may also negatively impact cell function. RAN translation products have been detected in the affected brain regions of patients, as well as in N171-82Q mice and a C. elegans model.18,19 However, the actual contribution of RAN translation products to HD is not clear, as, for example, no RAN toxicity was observed in HD140Q knock-in mice.20

The expanded polyQ-containing mutant HTT (mHTT) protein has been shown to interact aberrantly with a variety of proteins, including transcriptional regulators such as RNA polymerase II subunit A (POLR2A), Tumor protein p53, Mouse double minute 2 (MDM2), CREB-binding protein (CBP) and Heat shock protein 70 (HSP70), cell cycle regulators like Ras homolog enriched in brain (Rheb) and mammalian target of rapamycin (mTOR), and cytoskeleton proteins such as actin and neurofilament light (NF-L). These aberrant interactions result in a complex and widespread molecular pathology, affecting many essential processes in the cell, including DNA damage repair, transcriptional regulation, mitochondrial function and apoptosis.2125 Importantly, premature polyadenylation of the pre-mRNA as well as proteolytic cleavage of HTT protein lead to the production of a variety of HTT fragments, and there is ample evidence that such fragments, especially the short N-terminal species, are more toxic than the full-length mHTT protein.2635 In order to make tailored therapeutics towards the short toxic fragments, a good understanding of the mechanisms leading to their formation is needed. In this review, we therefore focus on how toxic N-terminal HTT protein species are produced and how they are linked to toxicity, as well as on therapeutic strategies that are capable of reducing these fragments.

N-terminal HTT protein fragments are mainly produced through two distinct processes: proteolytic cleavage and premature polyadenylation (see Figure 2 and Table 1).

Table 1 Overview of Proteolytic Cleavage Sites

Figure 2 Schematic overview of production of N-terminal HTT protein. (A) Regular splicing, overview of the resulting mRNA and full-length protein and the identified proteolytic cleavage sites. (B) Alternative splicing and premature polyadenylation and resulting transcript. (C) Resulting protein species and propensity for nuclear entry, aggregation and toxicity.

The group of Michael Hayden first showed that HTT could be cleaved proteolytically by apopain (caspase-3) in a repeat-length dependent manner.36 This was confirmed in a follow-up study, in which they mapped one of the caspase-3 cleavage sites to D513 and another site C-terminally of amino acid (aa) 548. Furthermore, two caspase-1 cleavage sites were identified in the first 548 aa. In contrast to their previous work with truncated HTT, the authors found no repeat-length dependence of cleavage efficiency of full-length HTT.37 In a third study, the authors were able to map the second caspase-3 cleavage site to D552, and further identified a caspase-6 cleavage site at D586.38 More recently, Martin et al recently identified yet another caspase cleavage site at D572, which was shown to be cleaved by caspase-1 and caspase-2.39

Both full-length and N-terminal caspase-cleavage products of HTT were found to be substrates for cleavage by calpains.4042 Four calpain cleavage sites have been mapped, at aa 437, 465/469 and 536/54041 and between aa 63111,42 calpain cleavage efficiency appears to be positively correlated with repeat length.41,42 Furthermore, it was shown that calpain levels, and in particular the active form, were increased in the caudate of HD patients compared to controls.41

Next to caspase and calpain generated fragments, various other cleaved HTT products have been described. Lunkes et al identified two N-terminal HTT fragments, cp-A and cp-B, which appeared to be generated in transfected NG108 cells through cleavage by aspartic endopeptidases. The C-terminus of HTT cp-A fragment was mapped between aa 104114. N-terminal fragments with the same immunogenic properties were identified in nuclear inclusions in post mortem frontal cortex of HD patients.43

Similarly, Schilling et al identified an N-terminal fragment ending between aa 90115 in post mortem tissues from HD patients and N171-82Q mice, as well as in transfected HEK293 cells.44 Further investigation in a HEK293 cell model revealed that short, HTT cp-B-like fragments were efficiently processed to HTT cp-A-like fragments, while longer HTT fragments proved to be inefficient substrates. The C-terminus of the HTT cp-A-like fragments was mapped between aa 105 and 115124. Although similar in size to the fragment described by Lunkes et al, inhibition of aspartyl proteases did not affect the formation of the cp-A-like fragment, and the authors were unable to identify any protease that generates these HTT cp-A-like fragments, suggesting that i) the fragments are not the same or ii) that the cp-A-like fragment described by Schilling et al is the same fragment but generated by a novel protease, which may be cell-type dependent.45 Ratovitski et al identified two N-terminal fragments (HTT cp-1 and cp-2) in PC12 and HEK293 cells expressing full-length HTT with 21Q or 126153Q or a truncated N1212 HTT fragment with 15Q or 138Q.46 These fragments were similar in size to the previously described HTT cp-A and cp-B fragments but were not affected by inhibition of aspartic endopeptidases. In addition, they were not affected by deletion of aa 105114. In combination with the epitope mapping, this narrowed the C-terminus of the HTT cp-1 fragment down to between aa 90 and 105, shorter than the cp-A and cp-A-like fragments described by Lunkes et al43 and Schilling et al44,45 Based on the absence of identified proteases and on the fragment length, we speculate that the generation of these fragments could involve aberrant splicing (see Aberrant Splicing and Premature Polyadenylation), although this would require further investigation.

Finally, Landles et al showed fourteen different N-terminal HTT protein isoforms (fragments 114) in brain tissue from HdhQ150 KI mice, the three shortest of which (fragments 1214) were specific to mHTT.33 Some of these fragments could be linked to specific proteolytic cleavage events: fragment 7 terminated at a novel calpain cleavage site between aa 510654, fragment 8 appeared to correspond to the D586 caspase-6 cleavage product, fragment 9 was likely produced by cleavage at calpain site 536 and fragment 10 by caspase cleavage at D513. Lastly, fragment 13 was determined to correspond to HTT-ex1.

In summary, many different proteases have been found to act on mHTT and wtHTT, generating N-terminal and C-terminal HTT fragments. The availability of antibodies that can recognize these fragments, as well as the possibility to specifically inhibit certain proteases, have allowed mapping of various fragments, albeit with variable resolution. Nonetheless, for multiple fragments, the mechanisms of production remain to be identified.

Besides proteolytic cleavage, there are other mechanisms that lead to the generation of toxic N-terminal mHTT fragments. Sathasivam et al showed that incomplete splicing of intron 1 leads to the production of a short premature polyadenylated HTT-ex1 transcript in various HD mouse models and that this HTT-ex1 can be translated into a 90 aa N-terminal HTT-ex1 protein (based on 23Q). HTT-ex1 transcript was also found to be expressed in HD patient fibroblasts and cortex.47 In a follow-up study, Neueder et al confirmed that the HTT-ex1 transcript can be detected in patient-derived fibroblasts, as well as HD patient cerebellum, sensory motor cortex and hippocampus, with the highest expression levels measured in juvenile HD patient tissues.48 The HTT-ex1 transcript has also been detected by RNA-sequencing in various HD mouse models, including BACHD, BAC-CAG and HdhQ111.17 Both in vitro and in patient-derived tissues, the production of the HTT-ex1 transcript appears to be positively correlated with CAG repeat length, showing much higher expression in cells and tissues derived from juvenile HD patients.48,49

The current hypothesis is that HTT-ex1 formation is influenced by a combination of sequestration of spliceosome components such as U1 snRNP at the CAG repeat, leading to less efficient splicing of exon 1 to exon 2, and a reduced transcription rate, which leads to longer exposure of the cryptic polyA site in intron 1. Although the Bates group initially found evidence for the involvement of Serine and Arginine Rich Splicing Factor 6 (SRSF6) in HTT-ex1 formation,47,49 they later found that the silencing of Srsf6 in HD mouse models did not affect HTT-ex1 formation.50 It has therefore been hypothesized that multiple RNA-binding proteins may be involved in the missplicing of HTT-ex1.12 Regardless of the exact mechanisms involved, aberrant mHTT splicing is CAG repeat length dependent, suggesting that HTT-ex1 formation and associated toxicity would increase as somatic instability progresses in HD48 and that interventions targeting repeat expansion and HTT-ex1 may have therapeutic advantage.

Consistently accumulating evidence indicates that small N-terminal fragments containing extended polyQ tracts significantly contribute mHTT cellular mislocalization, aggregation and toxicity. Initial studies by the Ross group showed that transfection of N2a or HEK293 cells with full-length HTT with either 23Q or 82Q, or of truncated HTT N171-18Q or N63-18Q resulted in a diffuse cytoplasmic localization of the protein. In contrast, transfection with N171-82Q or N63-82Q led to more punctate labeling in both cytoplasm and nucleus, with the short N63-82Q construct showing the most prominent nuclear localization.51 The Hayden group found similar results, showing that N-terminal fragments of 427, 548 or more aa formed mainly perinuclear aggregates, while fragments up to 224 aa showed both cytoplasmic and nuclear aggregates. Furthermore, they found that pathogenicity depended both on repeat length and on fragment size.26,27

Barbaro et al found that, in Drosophila, shorter N-terminal fragments were more toxic and more prone to aggregate, with HTT-ex1 being by far the most toxic species.28 In mice, the R6/2 model that expresses only HTT-ex1 is by far the most swiftly progressing HD mouse model,52,53 while conditional suppression of HTT-ex1 has been shown to be neuroprotective.54 Recent in vitro studies by the Lashuel group confirm these results and further extend the findings by showing that the polyQ and Nt17 domains of HTT-ex1 synergistically modulate the aggregation propensity of HTT-ex1, with a key role of the Nt17 domain in regulating HTT-ex1 aggregation dynamics and subcellular localization and toxicity.34

There is conflicting evidence with regard to the pathogenicity of nuclear and cytoplasmic mHTT. Some groups have reported evidence that nuclear localization is required for toxicity. For example, the Greenberg group showed that adding a nuclear export signal to a N171 HTT fragment blocked its toxicity in transfected striatal neurons.55 In contrast, the Hayden group reported that neither the addition of a nuclear localization signal to a N548 HTT fragment nor the addition of a nuclear export signal to a N151 fragment altered the toxicity of those fragments, suggesting that both the nucleus and the cytoplasm are sites of HD toxicity.56 Trushina et al found that nuclear entry of mHTT only occurred after commitment of a cell to cell death. Therefore, the authors argue that nuclear mHTT localization may not be the primary event leading to toxicity.57

Intranuclear and neuropil aggregates have been observed in most HD animal models,17,30,31,5863 and the presence of aggregates containing N-terminal HTT fragments has also been confirmed in patient brains by multiple groups.40,64,65 However, various groups have shown that it is not the insoluble aggregates or inclusion bodies, but rather the soluble oligomers that are the more toxic species.6669 In fact, some groups have found evidence that the formation of intranuclear inclusions may be protective,55,70,71 as reviewed by Arrasate and Finkbeiner.72 Mechanistically, this may be explained by the fact that soluble mHTT-ex1 oligomers have more aberrant protein interactions than insoluble aggregates and inclusions.73 Importantly, the length of N-terminal protein species and the associated sequence context, as well as post-translational modifications, also appear to play an important role in the aggregation process.35,74,75 For more in-depth reviews on the role of post-translational modifications, we redirect elsewhere.76,77

Various approaches have been investigated to therapeutically lower the expression or reduce the toxicity of the mutant HTT protein. The proteolytic cleavage pathway can be targeted to reduce the formation of N-terminal mHTT protein species. Furthermore, the N-terminal part of the protein can be targeted to reduce aggregation and/or increase clearance of mHTT. Finally, mHTT can be targeted at the transcript or gene level. Here, we will focus on approaches that are able to target not only full-length HTT but also HTT-ex1 and other N-terminal mHTT species, considering their potential therapeutic advantage (see Table 2).

Table 2 Overview of Studies Targeting HTT Protein

Caspase inhibition has been shown to reduce the proteolytic cleavage of mHTT and to improve the HD phenotype in BACHD78 and HdhQ111 mice.79 These results are backed up by earlier studies, where mutation of caspase-6 cleavage sites slowed down disease progression in YAC128 mice.80 However, it is not clear to what extent the protective effects are due specifically to the reduction of N-terminal mHTT species, rather than a general protective effect of caspase inhibition, as caspase inhibition was also protective in R6/2 and malonate models of HD, which do not express caspase-cleavable mHTT.8183

Using a different approach, Evers et al showed that removal of the caspase-6 cleavage site by antisense oligonucleotide (ASO)-mediated skipping of (part of) exon 12 led to reduced levels of the N568 fragment in vitro and in vivo in wild type and YAC128 mice.84,85 Except for the absence of astrogliosis, no data are available regarding phenotypic effects of this ASO treatment.

None of these approaches have yet successfully been translated into the clinic, and although all may potentially decrease the formation of toxic mHTT fragments and have the potential of allele-specificity, mechanisms of RNA-associated toxicity would not be addressed.

Aptamers are single-stranded oligonucleotides that, through their tertiary structure, can interact with target molecules such as proteins. The Roy lab identified aptamers that bind specifically to mHTT with 51 or 103Q but not wtHTT with 20Q.86,87 The selected aptamers were shown to inhibit aggregation of recombinant mHTT-ex1 in cell-free assays and in yeast, as well as reducing oxidative stress and mitochondrial dysfunction.86 To our knowledge, this approach has not yet been tested in vivo.

Various antibodies have been expressed intracellularly as intrabodies to target the N-terminus of HTT. In vivo, such intrabodies are delivered using viral vectors. An excellent review on the use of intrabodies in various neurodegenerative diseases was written by Messer and Butler.88

Two groups of intrabodies have been tested most extensively (see Figure 3): those that bind to the N-terminus of HTT (VL12.3, scFv-C4) and those that recognize the proline-rich regions (PRRs) in HTT-ex1 (MW7, Happ1, Happ3, INT41). In addition, there is some literature about polyQ-binding intrabodies (MW1, MW2) and a more C-terminal intrabody derived from EM48 (scFv-EM48).

Figure 3 Anti-HTT Exon 1 intrabodies. (A) Antigens used to select the published anti-HTT intrabodies. (B) Specific binding identified by crystallography for scFvC4 and VL12.3.

Notes: Reproduced from Messer A, Butler DC. Optimizing intracellular antibodies (intrabodies/nanobodies) to treat neurodegenerative disorders. Neurobiol Dis. 2020;134(October 2019):104619. doi: 10.1016/j.nbd.2019.104619 under Creative Commons BY-NC-ND 4.0.88

Southwell et al showed that intrabodies that bind to the PRR, ie, MW7, Happ1 and Happ3, increase the turnover of mHTT-ex1 overexpressed in vitro. VL12.3, an intrabody that binds to the N-terminal 17 aa of HTT, did not affect turnover, but did increase the nuclear localization of mHTT-ex1.90 In vivo, the PRR-binding Happ1 was shown to be beneficial in five different HD mouse models. In contrast, VL12.3, while effective in a lentiviral HD model, was ineffective in YAC128 mice and had a detrimental effect in R6/2 mice.91 The authors later showed that the increased turnover mediated by the PRR-binding intrabodies is dependent on a calpain-chaperone-mediated autophagy-dependent mechanism and that this process is blocked by VL12.3,92 explaining the detrimental effects of VL12.3.

Although scFv-C4 also binds to the N-terminus of HTT,93 its predominant cytoplasmic localization appears to protect from the detrimental effects observed for VL12.3.89 The scFv-C4 intrabody was shown to have beneficial effects in various HD models, including in vitro models, Drosophila and different mouse models.9498

Two additional intrabodies have been investigated: scFv-EM48 and INT41. Like Happ1, scFv-EM48, which binds just C-terminally to the second PRR, was shown to increase turnover of mHTT, and improved motor function of N171-82Q mice.99 INT41, an intrabody that recognizes the same epitope as Happ1, but which has enhanced cytoplasmic solubility, was shown to improve cognitive function in female R6/2 mice.100

In addition to the increased turnover induced by some of the intrabodies, the endogenous cellular machinery can be harnessed specifically to target proteins for degradation, using engineered proteins, peptides or small molecules. These can direct the protein of interest to the ubiquitin proteasome system, the autophagy-lysosomal pathway or chaperone-mediated autophagy. These approaches and their specific application in the context of HD have been extensively reviewed by Jarosiska and Rdiger.101

Two such approaches specifically target the polyQ region. Bauer et al engineered a fusion molecule consisting of two copies of a polyQ-binding peptide (QBP1) and heat shock-cognate protein 70 (HSC70)-binding motifs to induce chaperone-mediated autophagy.102 Clift et al co-expressed a polyQ-binding antibody (3B5H10) with TRIM21 in an approach that they call Trim-Away, to target mHTT for proteasomal degradation.103 Additionally, Butler et al produced a fusion protein consisting of the scFv-C4 intrabody and a PEST motif to enhance proteasomal degradation of HTT-ex1.104

Several endogenous proteins have been described to enhance the turnover of mHTT, including Praja1 ubiquitin ligase,105 TBK1106 and Blm10/PA200.107 Induction or overexpression of such proteins may represent a therapeutic strategy, although, so far, this notion is only supported by experiments in cellular, Drosophila, and C. elegans models. Additionally, specificity for mHTT has not been shown for any of these three proteins.

Finally, a small molecule that can bind to mHTT-ex1, called GLYN122, has been identified recently. GLYN122 was shown to reduce mHTT-ex1 aggregation in PC12 cells, as well as reducing mHTT in cortex and striatum of R6/2 mice after intraperitoneal injection.108

Next to targeting the pathogenic protein species itself, the production of such proteins can also be inhibited by targeting the HTT mRNA. Many different approaches have been tested to this effect, including ASOs, siRNAs, shRNAs and miRNAs (Table 3). Again, we only focus on those strategies that target HTT-ex1. Broadly speaking, the HTT-ex1 mRNA targeting approaches can be divided into those that target the expanded CAG repeat, and those that target other regions of HTT-ex1. In addition, some other approaches have been described.

Table 3 Overview of Studies That Evaluated Therapeutic Approaches Targeting HTT at the RNA Level

Many studies have tested ASOs or RNAi agents to target the CAG repeat.109122 In general, CAG-targeting confers preference towards the expanded allele, as this allows for binding of multiple molecules per mRNA.111 Only a few studies included in vivo efficacy. Yu et al showed the efficacy of their siRNA in HdhQ150 mice.115 Monteys et al used transgenic mice expressing tagged full-length wtHTT and mHTT, showing preferential silencing of mHTT.118 Datson et al showed the efficacy of their CAG-targeting ASO in R6/2 and Q175 mice,120 an ASO that is now further developed by Vico Therapeutics. Kotowska-Zimmer et al have shown that artificial miRNAs targeting the CAG repeat specifically reduced mHTT in YAC128 mice.122

A number of strategies that target other regions of HTT-ex1 have been described as well.123137 This approach would be expected to lower both wtHTT and mHTT. With the exception of Boado et al and Kordasiewicz et al, who used ASOs, all of these studies utilized RNAi agents. Various groups have demonstrated efficacy of siRNA or shRNA in R6/1, R6/2 and AAV100Q mice.127130 uniQures miRNA therapy has shown target engagement in the widest range of HD animal models, including Hu128/21, Q175 and R6/2 mice, lentiviral rat model and transgenic HD minipigs,123,124,132134,136 as well as a favorable safety profile in toxicity studies in rats and non-human primates.137

A handful of studies described other approaches to HTT RNA-targeting. Rindt et al developed a method to induce trans-splicing, by which mHTT exon 1 is replaced with exogenous wtHTT exon 1 in the mRNA. Thus far, there is only in vitro proof of principle for this approach, and the efficiency is rather low, with 1015% of trans-splicing observed even after extensive optimization.138,139 Batra et al have developed an RNA-targeting Cas9 approach which targets the CAG repeat.140 For HD, there is only in vitro evidence for this approach so far, but a similar approach targeting a CUG (cytosine, uracil, and guanine) repeat was shown to be effective in vivo in myotonic dystrophy type 1 mouse models.141 This platform is being developed by Locanabio.

Finally, some small molecules have been described to bind to either HTT-ex1 or the CAG repeat, most notably furamidine, myricetin and a series of pyridocoumarin derivatives, reviewed elsewhere.12 These compounds have been described to inhibit translation of HTT. However, specificity of such compounds is generally low, thereby increasing the chance of unwanted off-target effects.

Finally, several approaches that target the HTT gene have been described (Table 4).

Table 4 Overview of Studies Targeting the HTT Gene

Transcription can be prevented using zinc finger proteins (ZFPs) targeting the expanded CAG repeat.142144 This approach shows allele-selectivity for the expanded repeat and is currently being developed for the clinic by Sangamo and Takeda. Further, CRISPR-Cas9 genome editing approaches have been developed to either knock out HTT by inducing mutations or excise the region containing the CAG repeat. Several groups have shown in vitro and in vivo proof of principle using single guide RNAs directed to HTT-ex1 to induce HTT knockout.145148 Further, using a double guide RNA approach, various groups have shown that it is possible to excise the region containing CAG repeat.149154 The size of this region differs based on the chosen guide RNAs, with the first report by Shin et al deleting a large 44 kb region,149 while the most precise excision was shown by Yang et al and Monteys et al, who deleted only the CAG repeat and small flanking regions.150,151

Several HTT lowering therapies are either already in clinical trials or are close to entering the clinic. These therapies include different therapeutic modalities and mechanisms of action, each with distinct potential efficacy and safety profiles. Only the approaches in clinical trials or performing IND-enabling studies are covered here.

Two of the most advanced programs, the Phase III trial with the non-allele-specific HTT exon 36-targeting ASO tominersen (Roche) and the phase I/II trials with the allele-specific mHTT-associated single nucleotide polymorphism (SNP)-targeting ASOs WVE-120101 and WVE-120102 (Wave Life Sciences) were halted in 2021, as reviewed elsewhere.155 Roche plans to design a new Phase II study with tominersen, for younger adult patients with lower disease burden (https://ir.ionispharma.com/news-releases/news-release-details/ionis-partner-evaluate-tominersen-huntingtons-disease-new-phase). Wave Life Sciences has now initiated a new trial with their novel product WVE-003, which targets another SNP and has improved chemistry (clinicaltrials.gov NCT05032196). These ASOs are administered repeatedly through intrathecal administration, which may explain some of the adverse events observed with tominersen, which was more pronounced in the cohort receiving more frequent administration.155 Neither drug is expected to affect HTT-ex1 formation or RNA-mediated toxicity.

Novartis and PTC Therapeutics both have initiated Phase 2 clinical trials for their splicing modulators Branaplam (NCT05111249) and PTC518 (NCT05358717). These small molecules induce the inclusion of a pseudoexon between HTT exons 49 and 50, which leads to a premature stop codon and subsequent nonsense-mediated decay.156,157 One of the main advantages is that these small molecules can be administered orally. Furthermore, the mechanism of action targets the pre-mRNA and is therefore quite upstream in the molecular pathology. However, this approach is not specific for the mutant allele and, as it targets a downstream exon, is also not expected to affect HTT-ex1 production or toxic RNA gain-of-function.

In a more indirect fashion, metformin has been shown to reduce translation of HTT through interacting with the MID1/PP2A/mTOR protein complex.158 Interestingly, the effect of metformin was found to be specific for mHTT and to also impact HTT-ex1 protein formation. The drug can be administered orally, and as it is already in clinical use for the treatment of diabetes, its safety profile has already been well established. Metformin is currently being tested for the treatment of HD in a phase III clinical trial to establish its potential as a treatment for HD (NCT04826692). Although it has been shown to reduce HTT levels, RNA-mediated toxicity is not expected to be targeted by its mechanism of action.

There are no therapies that target HTT-ex1 exclusively, but some therapies target HTT-ex1 in addition to the full-length HTT. The most advanced is uniQures gene therapy AMT-130, which is currently being tested in phase I/II clinical trials (NCT04120493 and NCT05243017). AMT-130 is an AAV5-delivered miRNA which is administered through a one-time intrastriatal injection. This therapy is not allele-selective, and its effect on RNA-mediated toxicity has not yet been established.

Several other HTT-ex1 targeting candidates are close to entering clinical trials, including Galyan Bios HTT-ex1 binding small molecule GLYN122 and Vybions INT41 intrabody. These therapeutic candidates target the protein and are therefore not expected to impact RNA-mediated toxicity. According to the companies websites, both are performing IND-enabling studies, although their target date to enter the clinic is not clear (https://www.galyan.bio/pipeline, https://www.vybion.com/?page=product_pipeline).

Likewise, Vico Therapeutics received FDA orphan drug designation for their CAG-targeting ASO in July 2021 and is expected to start clinical trials soon (https://vicotx.com/pipeline/). Takeda and Sangamo are further developing their ZFP approach targeting the CAG repeat (https://www.sangamo.com/programs/). Both approaches preferentially target mHTT and as they act on the (pre-)mRNA and on transcription, respectively, these drug candidates may also have a beneficial effect on RNA-mediated toxicity.

Although all the approaches mentioned, as well as others in earlier phases of development, aim to reduce HTT levels, their mechanism of action is different and not all pathways related to HTT toxicity will be engaged. The relative contribution of each pathway is a matter of debate and is likely to depend on many factors, including age, tissue and cell type. Several of the described mechanisms of N-terminal HTT fragment production, including calpain cleavage and premature polyadenylation, have been shown to correlate with repeat length. This is also the case with HTT-ex1 formation through aberrant splicing. Therefore, it may be expected that as the repeat gets longer over time due to somatic instability, the contribution of these mechanisms will increase. Nonetheless, the broad molecular pathology of HD would likely benefit most from an intervention that acts as far upstream as possible, ie, on the DNA or the RNA level.

For an approach to be successful in disease modification, next to efficiency, adequate safety is key. Safety issues can arise from intrinsic characteristics of the therapeutic modality itself (eg, chemistry, properties of the therapeutic vector, and need of chronic administration), which are not covered in this review. The mechanism of action of the approach can also have different safety risks. Very specific approaches, with a well-understood mechanism, and with low to no interactions with other processes and molecules other than those related to HTT toxicity, would be preferred.

Multiple different approaches are running head-to-head. The small molecule splicing modulators are among the most elegant in terms of delivery, as these are capable of crossing the bloodbrain barrier and can therefore be administered orally. However, these small molecules are not specific for mHTT or even solely for HTT, and long-term studies are needed to determine the safety profile. Furthermore, these splicing modulators are expected to affect neither aberrant splicing of HTT-ex1 nor toxic RNA gain-of-function effects. ASOs and siRNAs have a less favorable distribution and need to be administered locally, although novel chemistries, such as peptide nucleic acids and di-siRNAs, have shown more promising biodistribution and may allow for systemic administration. These synthetic oligonucleotides are active for a limited amount of time, and therefore need to be readministered frequently. CAG-targeting ASOs are expected to not only reduce HTT and HTT-ex1 protein gain-of-function but also to alleviate RNA-mediated toxicity; however, non-specific effects on other genes containing CAG repeats may be difficult to overcome. Finally, the gene therapy approaches utilize AAVs to deliver their cargo. The current generation of AAVs is not sufficiently capable of crossing the bloodbrain barrier and therefore still needs to be administered locally, although efforts are ongoing to identify novel capsids that could be administered in a less invasive manner, eg, Goertsen et al.159 Because most cells that are targeted in HD are non-dividing, a more invasive route of administration is, however, less of an issue, as the therapy would only need to be administered once. uniQures miRNA-based strategy would reduce toxic protein gain-of-function, whereas Takeda and Sangamos ZFP approach targets DNA and thereby acts upstream of mHTT transcription, which would improve both toxic protein- and RNA gain-of-function; yet, as the mechanism of action of this approach involves direct targeting of the repeat, off-target effects may be an issue. Pre-clinically, gene editing approaches using CRISPR-Cas are being explored. However, long-term studies will need to show the safety profiles of such approaches.

To maximize therapeutic efficacy, future research will need to point out whether it may be advantageous to combine various therapeutic strategies with different modes of action. Further, it is likely that any therapeutic approach will benefit from as early intervention as possible. To this end, excellent safety profiles and good biomarkers of both safety and efficacy will be key.160

In summary, we have reviewed the production of N-terminal HTT protein fragments, their role in HD pathology, as well as therapeutic approaches to target these toxic species. Extensive research into HD continues to deepen our understanding of the broad molecular mechanisms leading to disease. With the increasing understanding of the pathological mechanisms associated with mHTT, several different therapeutic approaches are being developed, which will hopefully lead, in the near future, to halting or modification of this devastating disease.

We thank our uniQure colleagues who provided a critical review of the manuscript.

All authors made a significant contribution to the work reported, whether that is in the conception, study design, execution, acquisition of data, analysis and interpretation, or in all these areas; took part in drafting, revising or critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.

LB, ME and AV are employees of, and may own stock/options in, uniQure biopharma B.V. In addition, Dr Astrid Valls has a patent WO2021053018 issued to UNIQURE IP B.V. The authors report no other conflicts of interest in this work.

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35. Chongtham A, Bornemann DJ, Barbaro BA, et al. Effects of flanking sequences and cellular context on subcellular behavior and pathology of mutant HTT. Hum Mol Genet. 2020;29(4):674688. doi:10.1093/hmg/ddaa001

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Emerging Therapies for Huntington's Disease Focus on N-T | BTT - Dove Medical Press

REDWOOD CITY, Calif., Aug. 26, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced that Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies, will present at the Gilmartin Group Inaugural Emerging Growth Company Showcase, being held on Wednesday, August 31, 2022.

Interested parties can access a live webcast of the presentation directly by following this link. An archived webcast of the presentation will be available under the Events and Presentations in the Investors section of Adverums website.

About Adverum Biotechnologies

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures for these diseases to restore vision and prevent blindness. Leveraging the research capabilities of its proprietary, intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. By overcoming the challenges associated with current treatment paradigms for these debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit http://www.adverum.com.

Forward-looking Statements

Statements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; enrollment uncertainties; the results of early clinical trials not always being predictive of future clinical trials and results; and the potential for future complications or side effects in connection with use of Ixo-vec. Additional risks and uncertainties facing Adverum are set forth under the caption Risk Factors and elsewhere in Adverums Securities and Exchange Commission (SEC) filings and reports, including Adverums Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 filed with the SEC on August 11, 2022. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Corporate & Investor Inquiries

Anand ReddiVice President, Head of Corporate Strategy, External Affairs and EngagementAdverum Biotechnologies, Inc.T: 650-649-1358E: areddi@adverum.com

Media

Megan TalonAssociate Director, Corporate CommunicationsAdverum Biotechnologies, Inc.T: 650-649-1006E: mtalon@adverum.com

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Adverum to Present at the Inaugural Gilmartin Group Emerging Growth Company Showcase - GlobeNewswire

DUBLIN, Aug. 26, 2022 /PRNewswire/ -- The "Global Viral Vectors and Plasmid DNA Manufacturing Market (2022-2027) by Product Type, Application, Geography, Competitive Analysis and the Impact of Covid-19 with Ansoff Analysis" report has been added to ResearchAndMarkets.com's offering.

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The Global Viral Vectors and Plasmid DNA Manufacturing Market is estimated to be USD 901.01 Mn in 2022 and is expected to reach USD 2752.97 Mn by 2027, growing at a CAGR of 25.03%.

Market dynamics are forces that impact the prices and behaviors of the Global Viral Vectors and Plasmid DNA Manufacturing Market stakeholders. These forces create pricing signals which result from the changes in the supply and demand curves for a given product or service. Forces of Market Dynamics may be related to macro-economic and micro-economic factors.

There are dynamic market forces other than price, demand, and supply. Human emotions can also drive decisions, influence the market, and create price signals. As the market dynamics impact the supply and demand curves, decision-makers aim to determine the best way to use various financial tools to stem various strategies for speeding the growth and reducing the risks.

Company Profiles

The report provides a detailed analysis of the competitors in the market. It covers the financial performance analysis for the publicly listed companies in the market. The report also offers detailed information on the companies' recent development and competitive scenario. Some of the companies covered in this report are Merck KGaA, Lonza, FUJIFILM Diosynth Biotechnologies, Thermo Fisher Scientific, Cobra Biologics, Catalent, etc.

Countries Studied

America (Argentina, Brazil, Canada, Chile, Colombia, Mexico, Peru, United States, Rest of Americas)

Europe (Austria, Belgium, Denmark, Finland, France, Germany, Italy, Netherlands, Norway, Poland, Russia, Spain, Sweden, Switzerland, United Kingdom, Rest of Europe)

Middle-East and Africa (Egypt, Israel, Qatar, Saudi Arabia, South Africa, United Arab Emirates, Rest of MEA)

Asia-Pacific (Australia, Bangladesh, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Sri Lanka, Thailand, Taiwan, Rest of Asia-Pacific)

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Competitive Quadrant

The report includes Competitive Quadrant, a proprietary tool to analyze and evaluate the position of companies based on their Industry Position score and Market Performance score. The tool uses various factors for categorizing the players into four categories. Some of these factors considered for analysis are financial performance over the last 3 years, growth strategies, innovation score, new product launches, investments, growth in market share, etc.

Ansoff Analysis

The report presents a detailed Ansoff matrix analysis for the Global Viral Vectors and Plasmid DNA Manufacturing Market. Ansoff Matrix, also known as Product/Market Expansion Grid, is a strategic tool used to design strategies for the growth of the company. The matrix can be used to evaluate approaches in four strategies viz. Market Development, Market Penetration, Product Development and Diversification.

The matrix is also used for risk analysis to understand the risk involved with each approach. The analyst analyses the using the Ansoff Matrix to provide the best approaches a company can take to improve its market position. Based on the SWOT analysis conducted on the industry and industry players, the analyst has devised suitable strategies for market growth.

Why buy this report?

The report offers a comprehensive evaluation of the Global Viral Vectors and Plasmid DNA Manufacturing Market. The report includes in-depth qualitative analysis, verifiable data from authentic sources, and projections about market size. The projections are calculated using proven research methodologies.

The report has been compiled through extensive primary and secondary research. The primary research is done through interviews, surveys, and observation of renowned personnel in the industry.

The report includes an in-depth market analysis using Porter's 5 forces model and the Ansoff Matrix. In addition, the impact of Covid-19 on the market is also featured in the report.

The report also includes the regulatory scenario in the industry, which will help you make a well-informed decision. The report discusses major regulatory bodies and major rules and regulations imposed on this sector across various geographies.

The report also contains the competitive analysis using Positioning Quadrants, the analyst's Proprietary competitive positioning tool.

Key Topics Covered:

1 Report Description

2 Research Methodology

3 Executive Summary

4 Market Dynamics4.1 Drivers4.1.1 Increasing Capacities by Manufacturers Owing to Rising Demand4.1.2 Rise in Prevalence of Cancer, Viral Infections, and Genetic Disorders4.1.3 Increase in Awareness Regarding Gene Therapies4.2 Restraints4.2.1 High Cost Associated with Gene Therapies4.2.2 Stringent Government Regulations4.3 Opportunities4.3.1 The Rise in the Development of Allogeneic and Autologous Cell Therapy4.3.2 Increase in Funding for R&D Activities Pertaining to Gene Therapy4.4 Challenges4.4.1 Involved Risks For Mutagenesis and Other Obstruction in Gene Therapy

5 Market Analysis5.1 Regulatory Scenario5.2 Porter's Five Forces Analysis5.3 Impact of COVID-195.4 Ansoff Matrix Analysis

6 Global Viral Vectors and Plasmid DNA Manufacturing Market, By Product Type6.1 Introduction6.2 Plasmid DNA6.3 Viral Vector6.4 Non-viral Vector

7 Global Viral Vectors and Plasmid DNA Manufacturing Market, By Application7.1 Introduction7.2 Cancer7.3 Genetic Disorder7.4 Infectious Disease7.5 Other Applications

8 Americas' Viral Vectors and Plasmid DNA Manufacturing Market8.1 Introduction8.2 Argentina8.3 Brazil8.4 Canada8.5 Chile8.6 Colombia8.7 Mexico8.8 Peru8.9 United States8.10 Rest of Americas

9 Europe's Viral Vectors and Plasmid DNA Manufacturing Market9.1 Introduction9.2 Austria9.3 Belgium9.4 Denmark9.5 Finland9.6 France9.7 Germany9.8 Italy9.9 Netherlands9.10 Norway9.11 Poland9.12 Russia9.13 Spain9.14 Sweden9.15 Switzerland9.16 United Kingdom9.17 Rest of Europe

10 Middle East and Africa's Viral Vectors and Plasmid DNA Manufacturing Market10.1 Introduction10.2 Egypt10.3 Israel10.4 Qatar10.5 Saudi Arabia10.6 South Africa10.7 United Arab Emirates10.8 Rest of MEA

11 APAC's Viral Vectors and Plasmid DNA Manufacturing Market11.1 Introduction11.2 Australia11.3 Bangladesh11.4 China11.5 India11.6 Indonesia11.7 Japan11.8 Malaysia11.9 Philippines11.10 Singapore11.11 South Korea11.12 Sri Lanka11.13 Thailand11.14 Taiwan11.15 Rest of Asia-Pacific

12 Competitive Landscape12.1 Competitive Quadrant12.2 Market Share Analysis12.3 Strategic Initiatives12.3.1 M&A and Investments12.3.2 Partnerships and Collaborations12.3.3 Product Developments and Improvements

13 Company Profiles 13.1 Audentes Therapeutics13.2 Batavia Biosciences13.3 BioMarin Pharmaceutical13.4 BioNTech IMFS13.5 Catalent13.6 Cobra Biologics13.7 FUJIFILM Diosynth Biotechnologies13.8 Genezen laboratories13.9 Lonza13.10 Merck KGaA13.11 Miltenyi Biotec13.12 RegenxBio13.13 SIRION Biotech 13.14 Takara Bio13.15 Thermo Fisher Scientific13.16 Virovek 13.17 Waisman Biomanufacturing13.18 Wuxi Biologics

14 Appendix

For more information about this report visit https://www.researchandmarkets.com/r/n1d1m0

Media Contact:

Research and MarketsLaura Wood, Senior Managerpress@researchandmarkets.com

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Global Viral Vectors and Plasmid DNA Manufacturing Market (2022 to 2027) - Featuring Audentes Therapeutics, Batavia Biosciences and BioMarin...

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Market (5th Edition) report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035.

Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, LibmeldyT, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors).

Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain.

In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods.

For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics.

Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation.

Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

Key Questions Answered

Key Topics Covered:

1. PREFACE

2. EXECUTIVE SUMMARY

3. INTRODUCTION

3.1. Context and Background

3.2. Evolution of Gene Therapies

3.3. Classification of Gene Therapies

3.3.1. Somatic and Germline Gene Therapies

3.3.2. Ex Vivo and In Vivo Gene Therapies

3.4. Routes of Administration

3.5. Mechanism of Action

3.6. Overview of Gene Editing

3.6.1. Evolution of Genome Editing

3.6.2. Applications of Genome Editing

3.6.3. Available Genome Editing Techniques

3.7. Advantages and Disadvantages of Gene Therapies

3.7.1 Ethical and Social Concerns Related to Gene Therapies

3.7.2. Constraints and Challenges Related to Gene Therapies

3.7.3. Therapy Development Concerns

3.7.4. Manufacturing Concerns

3.7.5. Commercial Viability Concerns

4. GENE DELIVERY VECTORS

4.1. Chapter Overview

4.2. Viral and Non-Viral Methods of Gene Transfer

4.3. Viral Vectors for Genetically Modified Therapies

4.4. Types of Viral Vectors

4.5. Types of Non-Viral Vectors

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

5.1. Chapter Overview

5.2. Regulatory Guidelines in North America

5.3. Regulatory Guidelines in Europe

5.4. Regulatory Guidelines in Asia-Pacific

5.5. Reimbursement Scenario

5.6. Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW

6.1. Chapter Overview

6.2. Gene Therapy Market: Clinical and Commercial Pipeline

6.3. Gene Therapy Market: Early-Stage Pipeline

6.4. Gene Therapy Market: Special Drug Designations

6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)

7. COMPETITIVE LANDSCAPE

7.1. Chapter Overview

7.2. Gene Therapy Market: List of Developers

7.3. Key Players: Analysis by Number of Pipeline Candidates

8. MARKETED GENE THERAPIES

8.1. Chapter Overview

8.2. Gendicine (Shenzhen Sibiono GeneTech)

8.3. Oncorine (Shanghai Sunway Biotech)

8.4. Rexin-G (Epeius Biotechnologies)

8.5. Neovasculgen (Human Stem Cells Institute)

8.6. Imlygic (Amgen)

8.7. Strimvelis (Orchard Therapeutics)

8.8. LuxturnaT (Spark Therapeutics)

8.9. ZolgensmaT (Novartis)

8.10. Collategene (AnGes)

8.11. ZyntelgoT (bluebird bio)

8.12. LibmeldyT (Orchard Therapeutics)

9. KEY COMMERCIALIZATION STRATEGIES

9.1. Chapter Overview

9.2. Successful Drug Launch Strategy: ROOTS Framework

9.3. Successful Drug Launch Strategy: Product Differentiation

9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product

9.5. List of Currently Approved Gene Therapies

9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers

9.6.1. Strategies Adopted Before Therapy Approval

9.6.1.1. Participation in Global Events

9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms

9.6.1.3. Indication Expansion

9.6.2. Strategies Adopted During/Post Therapy Approval

9.6.2.1. Geographical Expansion

9.6.2.2. Participation in Global Events

9.6.2.3. Patience Assistance Programs

9.6.2.4. Awareness through Product Websites

9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms

9.7. Concluding Remarks

10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES

10.1. Chapter Overview

10.2. Lumevoq (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

10.3. OTL-103

10.4. PTC-AADC

10.5. BMN 270

10.6. rAd-IFN/Syn3

10.7. beti-cel

10.8. eli-cel

10.9. lovo-cel

10.10. SRP-9001

10.11. EB-101

10.12. ProstAtak

10.13. D-Fi

Continued here:
Gene Therapy Market Research Report 2022 - ResearchAndMarkets.com - Business Wire

Published: Published Date - 09:21 PM, Wed - 10 August 22

Hyderabad: This concept may seem quite fictional and even futuristic. However, this is what geneticists worldwide through gene therapy are pursuing, while trying to find cure for a wide range of diseases that challenge modern medicine including cancers, heart diseases, diabetes, haemophilia, AIDS, genetic disorders, among others.

Gene therapy involves altering the genes inside the cells of the human body, in order to treat or prevent the disease progression. Essentially, geneticists worldwide are exploring ways to utilise gene therapy to alter genetic composition of cells that are responsible for causing diseases and in the process find a long term cure for diseases. The potential to unlock the cure for a wide range of diseases has become a major driving force for researchers and pharma giants worldwide to focus their energies and resources on gene therapy.

So what exactly is gene and gene therapy?

The Gene Therapy Advisory and Evaluation Committee (GTAEC), which monitors clinical trials across India on gene therapies, defines Gene is the most basic and functional unit of heredity and inheritance and consists of a specific sequence of nucleotides in DNA or RNA located on chromosomes that encodes for specific proteins. The human genome comprises more than 20,000 genes. Gene therapy refers to the process of introduction, removal or change in content of an individuals genetic material with the goal of treating the disease and a possibility of achieving long term cure.

The genetic material that has to be introduced to the diseased cell is done through a vector, whch is usually a virus. Viruses are the preferred vectors or vehicles as they are adaptable and efficient in delivering genetic material, the GTAEC, said.

While worldwide major pharmaceutical companies are developing gene therapies for treatment of single gene defects like haemophilia and muscular dystrophy, the Department of Biotechnology (DBT), Government of India, Tata Memorial Hospital, Mumbai and IIT-Mumbai have collaborated to start clinical trials of gene therapy on cancer in India.

Gene therapy in cancer:

In the last few years, CAR- (Chimeric Antigen Receptor) T therapy, a form of gene therapy has emerged as a breakthrough treatment for cancer, especially for leukemia, lymphoma (cancer of the lymphatic system) and multiple myeloma or the cancer of the plasma cells.

The CAR-T cells are genetically engineered in a laboratory and they bind with the cancer cells and kill them. The therapy is available in a few cancer research centres (on clinical trials basis) in US and cost of treatment ranges anywhere from Rs 3 crore to Rs. 4 crore.

To reduce treatment costs, promote and support development CAR-T cell technology against cancers, for the first time in India, Biotechnology Industry Research Assistance Council (BIRAC), established by DBT, Tata Memorial Hospital and IIT Bombay, have launched clincal trials of CAR-T gene therapy to treat cancers. The CAR-T cells were designed and manufactured at Bioscience and Bioengineering (BSBE) department of IIT Bombay. The gene therapy study on cancers is in early phase clinical trials at Tata Memorial in Mumbai.

Regulation of gene therapy:

Realising the potential of gene therapies in treating complex diseases, the GOI is providing financial and even technical guidance to researchers through ICMR, DBT and DST. To ensure gene therapies are introduced in India and clinical trials for gene therapies are performed in an ethical, scientific and safe manner, the ICMR has also framed National Guidelines for Gene Therapy Product Development and Clinical Trials document.

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Health and Tech: The promise of gene therapy to cure cancers - Telangana Today

DUBLIN, Aug. 10, 2022 /PRNewswire/ -- The "Stem Cell Therapy Global Market Opportunities And Strategies To 2031" report has been added to ResearchAndMarkets.com's offering.

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The global stem cell therapy market reached a value of nearly $4,019.6 million in 2021, having increased at a compound annual growth rate (CAGR) of 70.9% since 2016. The market is expected to grow from $4,019.6 million in 2021 to $10,600.2 million in 2026 at a rate of 21.4%. The market is then expected to grow at a CAGR of 11.4% from 2026 and reach $18,175.4 million in 2031.

Growth in the historic period in the stem cell therapy market resulted from rising prevalence of chronic diseases, a rise in funding from governments and private organizations, rapid growth in emerging markets, an increase in investments in cell and gene therapies, surge in healthcare expenditure, and an increase in pharmaceutical R&D expenditure. The market was restrained by low healthcare access in developing countries, limited reimbursements, and ethical concerns related to the use of embryonic stem cells in the research and development.

Going forward, increasing government support, rapid increase in the aging population, rising research and development spending, and increasing healthcare expenditure will drive market growth. Factors that could hinder the growth of the market in the future include high cost of stem cell therapy, stringent regulations imposed by regulators, and high cost of storage of stem cells.

The stem cell therapy market is segmented by type into allogeneic stem cell therapy and autologous stem cell therapy. The autologous stem cell therapy segment was the largest segment of the stem cell therapy market segmented by type, accounting for 100% of the total in 2021.

The stem cell therapy market is also segmented by cell source into adult stem cells, induced pluripotent stem cells, and embryonic stem cells. The induced pluripotent stem cells was the largest segment of the stem cell therapy market segmented by cell source, accounting for 77.2% of the total in 2021. Going forward, the adult stem cells segment is expected to be the fastest growing segment in the stem cell therapy market segmented by cell source, at a CAGR of 21.7% during 2021-2026.

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The stem cell therapy market is also segmented by application into musculoskeletal disorders and wounds & injuries, cancer, autoimmune disorders, and others. The cancer segment was the largest segment of the stem cell therapy market segmented by application, accounting for 49.7% of the total in 2021. Going forward, musculoskeletal disorders and wounds & injuries segment is expected to be the fastest growing segment in the stem cell therapy market segmented by application, at a CAGR of 22.1% during 2021-2026.

The stem cell therapy market is also segmented by end-users into hospitals and clinics, research centers, and others. The hospitals and clinics segment was the largest segment of the stem cell therapy market segmented by end-users, accounting for 66.0% of the total in 2021. Going forward, hospitals and clinics segment is expected to be the fastest growing segment in the stem cell therapy market segmented by end-users, at a CAGR of 22.0% during 2021-2026.

Scope:

Markets Covered:

By Type: Allogeneic Stem Cell Therapy; Autologous Stem Cell Therapy

By Cell Source: Adult Stem Cells; Induced Pluripotent Stem Cells; Embryonic Stem Cells

By Application: Musculoskeletal Disorders and Wounds & Injuries; Cancer; Autoimmune Disorders; Others

By End-Users: Hospitals And Clinics; Research Centers; Others

Key Topics Covered:

1. Stem Cell Therapy Market Executive Summary

2. Table of Contents

3. List of Figures

4. List of Tables

5. Report Structure

6. Introduction

7. Stem Cell Therapy Market Characteristics

8. Stem Cell Therapy Trends And Strategies

9. Impact Of Covid-19 On Stem Cell Therapy Market

10. Global Stem Cell Therapy Market Size And Growth

11. Global Stem Cell Therapy Market Segmentation

12. Stem Cell Therapy Market, Regional And Country Analysis

13. Asia-Pacific Stem Cell Therapy Market

14. Western Europe Stem Cell Therapy Market

15. Eastern Europe Stem Cell Therapy Market

16. North America Stem Cell Therapy Market

17. South America Stem Cell Therapy Market

18. Middle East Stem Cell Therapy Market

19. Africa Stem Cell Therapy Market

20. Stem Cell Therapy Global Market Competitive Landscape

21. Stem Cell Therapy Market Pipeline Analysis

22. Key Mergers And Acquisitions In The Stem Cell Therapy Market

23. Stem Cell Therapy Market Opportunities And Strategies

24. Stem Cell Therapy Market, Conclusions And Recommendations

25. Appendix

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/5kc6gq

Media Contact:Research and MarketsLaura Wood, Senior Managerpress@researchandmarkets.comFor E.S.T Office Hours Call +1-917-300-0470For U.S./CAN Toll Free Call +1-800-526-8630For GMT Office Hours Call +353-1-416-8900U.S. Fax: 646-607-1907Fax (outside U.S.): +353-1-481-1716

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Stem Cell Therapy Global Market Report 2022: Rising Research and Development Spending, and Increasing Healthcare Expenditure Present Future...

When coronary arteries are blocked, starving the heart of blood, there are good medications and treatments to deploy, from statins to stents. Not so for heart failure, the leading factor involved in heart disease, the top cause of death worldwide.

Its whats on death certificates, said cardiologist Christine Seidman.

Seidman has long been interested in heart muscle disorders and their genetic drivers. She studies heart failure and other conditions that affect the myocardium the muscular tissue of the heart not the blood vessels where atherosclerosis and heart attacks come from, although their consequences are also felt in the myocardium, including heart failure.

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With her colleagues at Brigham and Womens Hospital and Harvard Medical School, she and a long list of international collaborators have been exploring the genetic underpinnings of heart failure. Based on experiments deploying a new technique called single-nucleus RNA sequencing on samples from heart patients, on Thursday they reported in Science their discovery of how genotypes change the way the heart functions.

Their work raises the possibility that some of the molecular pathways that lead to heart failure could be precisely targeted, in contrast to treating heart failure as a disease with only one final outcome.

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Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting, she said. To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

Seidman talked with STAT about the research, including how snRNAseq solves the smoothie problem, and what it might mean for patients. The conversation has been edited for clarity and brevity.

What happens in heart failure?

The heart becomes misshapen in one of two ways. It either becomes hypertrophied, where the walls of heart muscle become thickened and the volume within the heart is diminished, in what we call hypertrophic cardiomyopathy. Or it becomes dilated, when the volume in the heart is expanded and the walls become stretched. I think of it as an overinflated balloon, and that is called dilated cardiomyopathy.

Hypertrophy and dilatation are known to cause the heart over time to have profoundly diminished functional capacity. And clinically, we call that heart failure, much more commonly arising from dilated cardiomyopathy.

What does it feel like to patients?

When we see patients clinically, theyre short of breath, they have fluid retention. When we look at their hearts, we see that the pump function is diminished. That has led to a hypothesis of heart failure as sort of the end stage of many different disorders, but eventually the heart walks down a final common pathway. Then you need a transplant or a left ventricular assist device, or youre going to die prematurely.

What can be done?

Heart failure is a truly devastating condition, and it can arise early in life, in middle age, and in older people. There is no treatment for it, no cure for it, except cardiac transplantation, of course, which provides a whole host of other problems.

How did you approach this problem?

One of the questions we wanted to answer is, are there signals that we can discern that say there are different pathways and there are molecules that are functioning in those pathways that ultimately converge for failure, but through different strategies of your heart?

We treat every patient with heart failure with diuretics. We give them a series of different medications to reduce the pressure against which the heart has to contract. Im clinically a cardiologist, but molecularly Im a geneticist, so it doesnt make sense. If your house is falling down because the bricks are sticking together or if its falling down because the roof leaks and the water is pooling, you do things differently.

Tell me how you used single-cell RNA sequencing to learn more.

Looking at RNA molecules gives us a snapshot of how much a gene is active or inactive at a particular time point. Until recently, we couldnt do that in the heart because the approach had been to take heart tissue, grind it all up, and look at the RNAs that are up or down. But that gives you what we call a smoothie: Its all the different component cells those strawberries, blueberries, bananas mixed together.

But theres a technology now called single-cell RNA sequencing. And that says, what are the RNAs that are up or down in the cardiomyocytes as compared to the smooth muscle cells, as compared to the fibroblasts, all of which are in the cells? You get a much more precise look at whats changing in a different cell type. And thats the approach that we use, because cardiomyocytes [the cells in the heart that make it contract] are very large. Theyre at least three times bigger than other cells. We cant capture the single cell it literally does not fit through the microfluidic device. And so we sequenced the nuclei, which is where the RNA emanates from.

What did you find?

There were some similarities, but what was remarkable was the degree of differences that we saw in cardiomyocytes, in endothelial cells, in fibroblasts. Theres a signature thats telling us I walked down this pathway as compared to a different one that caused the heart to fail, but through activation or lack of activation of different signals along the way.

And that to me is the excitement, because if we can say that, we can then go back and say, OK, what happens if we were to have tweaked the pathway in this genotype and a different pathway in a different genotype? Thats really what precision therapy could be about, and thats where we aim to get to.

Whats the next step?

It may be that several genotypes will have more similarities as compared to other genotypes. But understanding that, I think, will allow us to test in experimental models, largely in mice, but increasingly in cellular models of disease, in iPS [induced pluripotent stem] cells that we can now begin to use molecular technologies to silence a pathway and see what that does to the cardiomyocytes, or silence the fibroblast molecule and see what that does in that particular genotype.

To my mind, thats the way to drive precision therapeutics. We know the cause of heart failure. We intervene in a pathway that we know is activated. And for the first time, we have that information now from human samples, not from an experimental model.

What might this mean for patients?

If we knew that an intervention would make a difference thats where the experiments are we would intervene when we saw manifestations of disease. So the reason I can tell you with confidence that certain genes cause dilated cardiomyopathy is theres a long time between the onset of that expansion of the ventricle until you develop heart failure. So theres years for us to be able to stop it in its tracks or potentially revert the pathology, if we can do that.

What else can you say?

I would be foolish not to mention the genetic cause of dilated cardiomyopathy. Ultimately, if you know the genetic cause of dilated cardiomyopathy, this is where gene therapy may be the ultimate cure. Were not there yet, but we certainly have the capacity to make small molecules to interfere with pathways that we think are deleterious to the heart in this setting.

My colleagues have estimated that approximately 1 in 250 to 1 in 500 people may have an important genetic driver of heart muscle disease, cardiomyopathy. Thats a huge number, but not all of them will progress to heart failure, thank goodness. Around the world, there are 23 million people with heart failure. Its what ends up on most peoples death certificate. It is the most common cause of death.

Its a huge, huge burden. And there really is no cure for it except transplantation. We dont have a reparative capacity, so were going to have to know a cause and be able to intervene precisely for that cause.

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New research digs into the genetic drivers of heart failure, with an eye to precision treatments - STAT

New York, Aug. 08, 2022 (GLOBE NEWSWIRE) -- The global intracranial therapeutic delivery market is currently valued at around US$ 1.6 Bn and is anticipated to progress at an impressive CAGR of 7.9% over the 2022-2032 study period.

Cell and gene therapies are at the forefront of innovation in treating severe diseases, such as cancer, as well as rare diseases, accounting for around 12 percent of the pharmaceutical industrys clinical pipeline. However, the growing focus on effective therapy has impacted positive financial grades for cell and gene therapy throughout the clinical and social spectrum; intracranial therapeutic administration has been gaining favor in the biopharma industry.

The progressive development of CRISPR and next-generation sequencing has led to a surge in the interest in gene therapy and cell treatment in the past few years. The manufacturing community for cell and gene therapies, including pharmaceutical companies, contract development and manufacturing organizations (CDMOs), and suppliers of lab supplies and equipment, are looking into ways to strengthen supply chains and address process bottlenecks.

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Sales footprint expansion, which has been gaining more and more traction among key participants, calls for the desired assistance, based on financial approvals and consolidated activities. Additionally, several clinical trials have been carried out in association with research institutes.

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Rising prevalence of neurological disorders and increasing research activities for the development of regenerative medicine to drive market growth over the coming years, says an analyst of Persistence Market Research.

Market Competition

The therapeutic delivery for intracranial is a highly consolidated market with limited key manufacturers operating in the industry. A majority of market players are focused on offering a limited range of cell, gene, and enzyme replacement therapy used for neurological disorder indications.

To strengthen their position in the global market, key players are focusing on strategic approaches such as mergers and collaborations to improve their production capabilities and expand their portfolios in various clinical and research fields.

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Persistence Market Research offers a unique perspective and actionable insights on the intracranial therapeutic delivery market in its latest study, presenting a historical demand assessment of 2017 2021 and projections for 2022 2032.

The research study is based on the therapy (cell-based therapy, gene therapy, and enzyme replacement therapy) and indication (spinal muscular atrophy (SMA), multiple sclerosis, batten disease), and amyotrophic lateral sclerosis, across three key regions of the world considered in the taxonomy.

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The Alliance for Regenerative Medicine Announces the Appointment of Timothy D. Hunt as Chief Executive Officer

WASHINGTON, D.C. August 10, 2022

The Alliance for Regenerative Medicine (ARM), the leading international advocacy organization representing the cell and gene therapy sector, today formally announced that its Board of Directors has appointed Timothy D. Hunt as the organizations next Chief Executive Officer. Hunt will succeed Janet Lynch Lambert, who announced in April her plan to step down as CEO and who served on the Boards Search Committee. Hunt will start at ARM on September 6.

We are excited to welcome Tim to the ARM team at such a pivotal moment for our sector, said Emile Nuwaysir, Chair of the ARM Board and Search Committee,and President and Chief Executive Officer of Ensoma, an in vivo genomic medicines company. Tims two decades of experience advocating for biotechnology companies, knowledge of the key issues facing the cell and gene therapy field, and expertise in leading teams make him the ideal choice to guide ARM in building the future of medicine. Tim has a deep philosophy of engagement with major stakeholders that will support ARM members and help bring cell and gene therapies into mainstream medical practice.

Hunt was most recently the Chief Culture and Corporate Affairs Officer at Xilio Therapeutics, a biotechnology company developing tumor-selective immuno-oncology therapies for patients with cancer. Prior to that, he was the Chief Corporate Affairs Officer at CRISPR gene-editing pioneer Editas Medicine, where he led the companys global policy and government affairs, bioethics, communications, market development and human resources initiatives. He also served in executive public affairs roles at Cubist Pharmaceuticals and Biogen.

Hunt was an Advisory Group member of the Value-Based Payments for Medical Products consortium at the Duke-Margolis Center for Health Policy. He also has been a member of the Board of Directors of the non-profit organization Life Science Cares and has chaired the Ethics Committee of the American Society of Gene and Cell Therapy (ASGCT). Hunt previously served as a member of ARMs Gene Editing Task Force and on the Biotechnology Innovation Organizations Gene Editing Working Group. He received a B.A. in history and philosophy from Boston College and a J.D. from the Columbus School of Law at the Catholic University of America.

I am honored to succeed Janet as Chief Executive Officer of the Alliance for Regenerative Medicine and for the tremendous opportunity to build upon her legacy of developing ARM into the leading sector advocate and resource for the industry, said Hunt. Cell and gene therapies are already transforming patients lives, and we are on the cusp of even more breakthroughs in both rare and prevalent diseases. Our mission is both urgent and clear: to engage all our major stakeholders to ensure the patients we serve have access to the durable and potentially curative therapies of the present and future.

Tim is an excellent choice to continue to grow and strengthen this amazing organization and help realize the potential of regenerative medicine, said Lambert, whose tenure includes doubling ARMs global membership to 425 members, strengthening the organizations advocacy in the US and Europe, and building the ARM team.

Cell and gene therapies to treat blood cancers, spinal muscular atrophy, and an inherited form of blindness are approved in the US and Europe. 2022 could be a record year for new gene therapy approvals for rare disease, and regulators in the US and Europe could approve the first such therapies for hemophilia and sickle cell disease in late 2022 and 2023. More than 2,400 regenerative medicine clinical trials 60% of which targeted prevalent diseases including diabetes and cardiovascular disease were active globally at the end of 2021. ARM is committed to working with stakeholders to ensure that patients benefit from this rapidly advancing pipeline of transformative therapies.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organisation dedicated to realizing the promise of advanced therapy medicinal products (ATMPs).ARMpromotes legislative, regulatory, reimbursement and manufacturing initiativesin Europe and internationally to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-engineered therapies.Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing ATMPs.In its 12-year history,ARMhas become the global voice of the sector, representing the interests of 425+ members worldwide and 80+ members across 15 Europeancountries, including small and large companies, academic research institutions, major medical centres and patient groups.To learn more aboutARMor to become a member, visithttp://www.alliancerm.org.

Media inquiries

For more information or for media requests, please contact Stephen Majors, Senior Director of Public Affairs for ARM, atsmajors@alliancerm.org.

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The Alliance for Regenerative Medicine Announces the Appointment of Timothy D. Hunt as Chief Executive Officer - GlobeNewswire

COVID-19 came with plenty of mysteries, some of which have been resolved, and many that haven't. One mystery since the beginning of the pandemic was why children seemed much less susceptible to the virus than adults. For that and more research news, continue reading.

Why Kids May Have More Protection Against COVID-19

Generally, children are less at risk of COVID-19 infection than adults - not immune, but less likely to be infected and less likely to have severe disease. But why?

Researchers at theUniversity of Queensland, Australia,suggestthat children's nasal lining, or the nasal epithelium, inhibits infection and replication, particularly of the original Wuhan wild-type strain of SARS-CoV-2 and the Delta variant. However, the Omicron variant and its subvariants do not seem to have the same issue. They published their research inPLOS Biology.

"We have provided the first experimental evidence that the pediatric nasal epithelium may play an important role in reducing the susceptibility of children to SARS-CoV-2," the authors wrote. "The data strongly suggest that the nasal epithelium of children is distinct and that it may afford children some level of protection from ancestral SARS-CoV-2."

They found that the wild-type virus replicated less efficiently and was linked to an increased antiviral response in the nasal epithelial cells of children. The effect was also seen with the Delta variant, but not with the Omicron. They also found that Omicron replicated better in pediatric nasal epithelial cells than the wildtype or Delta, although they caution that this was a small study and may reflect how the virus is evolving.

New Molecules Discovered for Treating Age-Related Diseases

Researchers at theHebrew University of Jerusalemhaveidentifieda group of molecules that help cells repair damaged components. Impaired mitophagy is associated with various aging-associated diseases, such as Alzheimer's and Parkinson's, as well as sarcopenia.

Augmenting mitophagy, where defective mitochondria are removed and replaced by new ones, is an emerging strategy for treating the elderly. The researchers designed and tested a family of compounds related to spermidine, a known mitophagy-promoting agent. A prototype, 1,8-diaminooctane (VL-004), was better than spermidine in its ability to induce mitophagy and protect against oxidative stress.

The researchwas publishedin the journalAutophagy. The researchers founded a biotech company, Vitalunga, that is working to develop the drug.

Unique Subset of Immune Cells Protects Against Stroke

A study from theUniversity of Pittsburgfounda subset of white blood cells, a novel subset of CD8+ regulatory-like T cells (CD8+TRLs), that appear to confer fast-acting and lasting protection against ischemic stroke in mice.

These cells were attracted to the site of ischemic injury by a unique "homing" signal produced by the damaged brain cells. The CD8+TRLs reached the brain 24 hours after the stroke, then released molecules that provide direct neuroprotective effects and knock down inflammation and secondary brain damage.

In the mice studies, the animals who received a transfusion of purified CD8+TRLs did better and recovered faster than the untreated mice over five weeks. The CD8+TRLs appear to act as early responders to alert and recruit defenses after a stroke.

Gene Therapy Shows Promise in ALS

Researchers at theUniversity of California San DiegoSchool of Medicinereportthat gene therapy in animal models of ALS measurably delayed disease onset. Although most ALS cases are of unknown cause, the mouse and rat models were of an inherited form of ALS.

The therapy involved an AAV-vector carrying synapsin-Caveolin-1 cDNA into the spinal cords of the mice. The SynCav1 protected and preserved spinal cord motor neurons and added to the mice's longevity. They speculate that SynCav1 might serve as novel gene therapy for ALS and other forms of CNS disease.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease marked by progressive loss of motor neurons in the brain and spinal cord. It is fatal, and there are no cures.

New Therapeutic Target for Colorectal Tumors

Researchers at theTokyo University of Scienceidentifieda potential new therapeutic target for colorectal tumors.

Inflammatory bowel disease (IBD), which includes Crohn's disease and ulcerative colitis, can often lead to colorectal tumors. Innate immune receptors, such as C-type lectin receptors (CLRs), are responsible for the development of IBD. But they also play a key role in regulating gut microbiota and defending against pathogens.

One of those CLRs is a dendritic cell immunoreceptor (DCIR), responsible for maintaining the homeostasis of the immune and skeletal systems. Working with mice, the researchers found that mice without DCIR had less colitis severity and AOM-DSS-induced colorectal tumor growth. They found that using an antibody called anti-NA2 against asialo-biantennary-N-glycans (NA2), which binds to DCIR, decreased colitis symptoms and prevented colorectal tumor growth.

Two Common Viruses Associated with Alzheimer's Disease

Astudyout ofTufts Universityand theUniversity of Oxfordstrengthened the association between specific viral infections and Alzheimer's disease. Two common viruses can lie dormant in brain cells herpes simplex virus (HSV) and varicella-zoster virus (VZV); the latter is associated with shingles and chickenpox.

Researchers leveraged 3D human tissue culture models to mimic the brain and demonstrated that VZV may activate HSV to lay the groundwork for the early stages of Alzheimer's disease. HSV-1, the primary variant of HSV, when activated, leads to the accumulation of tau and amyloid-beta proteins, which are implicated in Alzheimer's disease. They published their research in theJournal of Alzheimer's Disease.

"Our results suggest one pathway to Alzheimer's disease, caused by a VZV infection which creates inflammatory triggers that awaken HSV in the brain," Dana Cairns, Ph.D., GBS12, a research associate in the Biomedical Engineering Department at Tufts, said. "While we demonstrated a link between VZV and HSV-1 activation, it's possible that other inflammatory events in the brain could also awaken HSV-1 and lead to Alzheimer's Disease."

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Research Roundup: Why Kids Have More Protection Against COVID-19 & More - BioSpace

According toGrand View Research, the global cell therapy market was valued at $7.8 billion in 2020 and is expected to expand at a compound annual growth rate (CAGR) of 14.5% between 2021 and 2028.

The rising number of clinical studies for cell-based therapies and investments in the industry may have a symbiotic relationship. The industry is seeing a snowballing number of ongoingclinical trialswith funding from governments and private agencies.

Theres an arguably thin line between cell and gene therapy. Cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease, according to theAmerican Society of Gene and Cell Therapy (ASGCT). The cells may originate from the patient (autologous cells) or a donor (allogeneic cells).

Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. Some protocols use both gene therapy and cell therapy.

Companies are using thebuilding blocks of lifeand advanced technologies to improve the treatment of human diseases and disorders such as cancer, providing an alternative to traditionally relied-on drugs and surgical treatments.

Cell therapy companies like Longeveron Inc. LGVN, Biogen Inc. BIIB, Alzamend Neuro Inc. ALZN and Solid Biosciences Inc. SLDB, as a result, have gained attention for their progress in using living cells to treat previously incurable diseases and disorders.

COVID-19 has reportedly causedsignificant disruptionto the cell and gene therapy industry. The pandemic has exacerbated the woes of an industry thats had its fair share of challenges with the supply of materials and the manufacturing and logistics processes.

General investments also slowed for the industry as governments shifted focus to saving lives and reviving economies. But things are starting to pick up now that the pandemic is on a downward trend.

Regulatory bodies like the Food and Drug Administration (FDA) have been urged to be more flexible in their approval timelines to make therapies affordable. Discussions continue around access and ensuring these therapies are affordable, reimbursable and profitable for the biopharmaceutical companies that develop them.

Academic and industry collaborations are expected to continue to expand and grow with noticeable impacts on the approval of products. Partnerships among academia, global pharmaceutical companies and small biotechs are expected to continue to shape the cell and gene therapy industry.

Longeveron, a clinical-stage biotechnology company, is one example of a company in the industry that has seemingly done well even during the pandemic. The company reports developing cellular therapies for investigation in chronic aging-related and certain life-threatening conditions.

The companys lead investigational product is Lomecel-B, a cell-based therapy product, derived from culture-expanded medicinal signaling cells sourced from the bone marrow of young, healthy adult donors.

Longeveron believes using the same cells that promote formation of new blood vessels, enhance cell survival and proliferation, inhibit cell death, and modulate immune system function may result in safe and effective therapies for some of the most difficult disorders associated with aging and some medical disorders.

Longeveron is sponsoring Phase 1 and 2 clinical trials in the following indications: Aging frailty, Alzheimers disease, metabolic syndrome, acute respiratory distress syndrome and hypoplastic left heart syndrome.

The companys mission is to advance Lomecel-B and other cell-based product candidates into pivotal Phase 3 trials to achieve regulatory approvals, subsequent commercialization and broad use by the healthcare community.

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Is This Company In A Special Position Even As The COVID-19 Pandemic Affects Cell-Based Therapy Industry? - Benzinga

OHSU researchers will receive a grant to helpadvance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos. (OHSU/Christine Torres Hicks)

Scientists at Oregon Health & Science University have received significant philanthropic support to advance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos.

The technique, initially demonstrated in mice, could eventually provide a new avenue for child-bearing among couples unable to produce viable eggs of their own.

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, andShoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. (OHSU/Christine Torres Hicks)

Even though the proof of concept in mice shows promise, significant challenges remain to be resolved before the technique could be ready for clinical trials under strict ethical and scientific oversight. Even then, Congress currently precludes the Food and Drug Administration from providing oversight for clinical trials involving genetic modification of human embryos.

Shoukhrat Mitalipov, Ph.D., (OHSU)

It will take probably a decade before we can say were ready, said Shoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. The science behind it is complex, but we think were on the right path.

This type of research is not funded by the National Institutes of Health, so it depends on philanthropic support. For this project, Open Philanthropy awarded $4 million over three years through the OHSU Foundation.

Paula Amato, M.D. (OHSU)

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, sees the potential for an enormous benefit to families struggling to have children if the technique proves successful.

Age-related decline in fertility remains an intractable problem in our field, especially as women are delaying childbearing, said Amato, who is the principal investigator for the grant award.

The technique holds promise for helping families to have genetically related children, a cohort that includes women unable to produce viable eggs because of age or other causes, including previous treatment for cancer. It also raises the possibility of men in same-sex relationships having children genetically related to both partners.

The skin cell can come from somebody who doesnt have any eggs themselves, Amato said. The biggest implication is for female, age-related infertility. It can also come from women with premature ovarian insufficiency due to cancer treatment or genetic conditions, or from men who would be able to produce a genetically related child with a male partner.

The award from Open Philanthropy will enable OHSU researchers to develop the technique in early human embryos using eggs and sperm from research donors. As with other groundbreaking research at OHSU including a gene-editing discovery that generated worldwide attention in 2017 none of the early embryos will be allowed to develop past the early blastocyst stage.

Researchers will build on a study in mice published this January in the journal Communications Biology.

The study demonstrated that it is possible to produce normal eggs by transplanting skin-cell nuclei into donor eggs from which the nuclei have been removed. Known as somatic cell nuclear transfer, the technique was famously used in 1997 to clone a sheep in Scotland named Dolly. In contrast to a direct clone of one parent, the mouse study published earlier this year required OHSU and collaborating scientists to cut the donor DNA in half and then fertilize the resulting egg with sperm to generate a viable embryo with chromosomes from both parents.

The process involves implanting the skin cell nuclei into a donor egg, and then allowing the egg to discard half its skin cell chromosomes a process similar to meiosis, when cells divide to produce sperm or egg cells. This results in a haploid egg with a single set of chromosomes with precisely half the chromosomes of the diploid skin cell with two sets of chromosomes. At just the right phase of the cell cycle, the new egg is combined with sperm chromosomes through in vitro fertilization.

An embryo then develops with the correct diploid number of chromosomes from each parent.

We had to show in the mouse that this hypothesis works, Mitalipov said. Open Philanthropy saw the implications for fertility with a new way of looking into this. The key is inducing haploidy.

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The new report titled Hemophilia Gene Therapy Market offer by Key Players, Types, Applications, Countries, Market Size, Forecast to 2030offered by Market Research, Inc. includes a comprehensive analysis of the market size, geographical landscape along with the revenue estimation of the industry. In addition, the report also highlights the challenges impeding market growth and expansion strategies employed by leading companies in the Hemophilia Gene Therapy Market.

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia A, B, and C. Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage.

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This market study covers and analyzes the potential of the global Hemophilia Gene Therapy industry, providing geometric information about market dynamics, growth factors, major challenges, PEST analysis and market entry strategy analysis, opportunities and forecasts. One of the major highpoints of the report is to provide companies in the industry with a strategic analysis of the impact of COVID-19 on Hemophilia Gene Therapy market.

Hemophilia Gene Therapy Market: Competition Landscape

The Hemophilia Gene Therapy market report includes information on the product presentations, sustainability and prospects of leading player including: BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo Therapeutics, Inc., and Freeline Therapeutics

Hemophilia Gene Therapy Market: Segmentation

By Type:

By Application:

Hemophilia Gene Therapy Market: Regional Analysis

All the regional segmentation has been studied based on recent and future trends and the market is forecasted throughout the prediction period. The countries covered in the regional analysis of the Global Hemophilia Gene Therapy market report are North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and Latin America.

Years Considered for the Hemophilia Gene Therapy Market Size:

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Major Points Covered in TOC:

Market Summary: It incorporates six sections, research scope, major players covered, market segments by type, Hemophilia Gene Therapy market segments by application, study goals and years considered.

Market Landscape: Here, the global Hemophilia Gene Therapy Market is dissected, by value, income, volume, market rate, and most recent patterns. The development and consolidation of the overall industry and top organizations is provided through graphs and piece of the pie for organizations.

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Market Status and Outlook by Region: In this segment, the report examines the net deals, income, creation and portion of the overall industry, CAGR and market size by locale. The global Hemophilia Gene Therapy Market is profoundly examined based on areas and nations like North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

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Market Forecasts: In this section, accurate and validated values of the total market size in terms of value and volume are provided by the research analysts. Also, the report includes production, consumption, sales, and other forecasts for the global Hemophilia Gene Therapy Market.

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Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

An FDA investigation revealed that the principal investigator and/or the team running the gene therapy trial at the University of Pennsylvania failed to disclose that before Gelsinger was treated, other patients had experienced alarming side effects and that monkeys administered the same engineered virus had died. The incident, coupled with dangerous outcomes involving other gene therapy treatments, had a chilling effect on the field and investors backed away.

Today, new approaches to gene therapy that include advances driven by CRISPR gene editing tools are raising hopes of a gene therapy revival. There are potential breakthroughs in the pipeline, including treatments for different types of cancer and sickle cell disease.

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Im concerned that gene therapy 2.0 is at risk of making the same mistakes that plagued the 1.0 version. Most notably, exciting work to translate gene therapy advances into safe, effective, and commercially viable treatments are at risk of being undermined by a reluctance to share data.

Im not saying that the field is on the verge of something analogous to the tragedy experienced in 1999. But the road to such extremes can be paved with a series of lesser nondisclosures that inhibit the free flow of scientific data essential for assessing potential risks long before treatments are given to people.

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I saw an example in May at the annual meeting of the American Society of Gene and Cell Therapy. A presenter was discussing a new approach for using gene therapy to treat a rare genetic disorder called Leber congenital amaurosis that causes blindness in children. The presenter discussed experiments in mice that involved disabling or deleting certain segments of a gene linked to the disorder, and this treatment appeared to at least slow the process of vision loss.

Assessing the safety and effectiveness of this potentially exciting advance requires knowing which regions of the gene were being modified. But when someone in the audience asked for more details, the presenter indicated that the information was proprietary and he would not disclose that. The exasperated questioner pointed out that the presenters approach was all about deleting [a segment of a gene], but you dont say what you are deleting. The response? Silence.

Tinkering with a particular genes function, even when the goal is to stop it from doing harmful things, can be risky. Knowing which region of a gene is being altered is essential for determining if solving one problem might create an even bigger one. For example, I and other scientists are interested in the potential of using CRISPR gene editing tools to disable a gene called nuclear factor erythroid 2-related factor 2 (NRF2), which produces a protein that protects a certain type of lung cancer tumor from the effects of chemotherapy or radiation. But that same gene confers a range of health benefits, such as withstanding toxic insults like nicotine or radiation or heat stress, and a poorly targeted edit might do more harm than good.

I am also regularly seeing a failure to disclose important details, like studies that use several strains of mice in a gene therapy experiment without clarifying which strain was linked to a particular result. That can make it difficult, or even impossible, to conduct a fundamental exercise in science: reproduce the results of other investigators. Reproducibility is a problem across all areas of science. But the stakes are higher in the fragile world of gene therapy, where investigators need to be doing all they can to protect patient safety.

My initial thoughts about the presenter who was loquacious in describing his success with gene therapy for Leber congenital amaurosis but quiet about revealing his methods was simply, If you arent willing to share your full information, dont present at scientific conferences.

I am not against protecting intellectual property or patenting biotechnology advances. Properly used, these strategies help attract investment that accelerates the search for transformative treatments. But researchers shouldnt come to a place where they benefit from everyone else freely sharing their findings but then refuse to reciprocate.

My broader message is that the field of gene therapy was once severely damaged by not sharing data that may have been able to prevent a young man from losing his life. Transparency in gene therapy research which can be accomplished without compromising commercial prospects is vital to success. One high-profile failure would badly hurt the revival of gene therapy; two would send it back into hibernation.

When everyone embraces transparency, all of our projects are likely to advance faster toward safe and effective treatments and everyone wins: scientists, investors and, most importantly, the people who could benefit from gene therapy.

Eric B. Kmiec is the executive director and chief scientific officer of the ChristianaCare Gene Editing Institute in Newark, Del.

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Secrecy: A demon of gene therapy's past bedevils its future - STAT

Recently, a patient centricity concept has spread across all corners of the healthcare community with a straightforward goal: to improve and save patients lives through innovative and effective drug therapies adapted to patients' individual needs. However, for this concept to truly come to life, it is necessary to count on temperature-controlled supply chain logisticslike CRYOPDP to turn what could be a complex operation into a highly efficient process. This is extremely vital, because a patients life could literally be on the line with every shipment.Patient centricity in the clinical trial community involves meeting the patients where they are.The clinical trial industry is globalising and demands international healthcare logistics partners that can serve its growing and continuously evolving needs. For decades, temperature-controlled logistics experts have operated behind the scenes with patients barely aware of the hard work that went into their medicines journey. Thanks to the Covid-19 vaccine distributions impressive performance and its universal effect on people's health across the globe, it has become apparent to the world just how critical and important a logistics providers mission is for both patients and the industry.The endless pandemic lockdowns meant that patients could not travel to hospital sites for clinical trials due to travel and access restrictions. This led to a seismic shift where sponsors quickly moved to a direct-to-patient model to try to continue trials and keep patient treatments on schedule. As part of our response to this unique situation, CRYOPDP as a specialist in temperature-controlled logistics, with the mission to improve people's healthcare options, had to go further and dive deeper to better understand customers perceptions of the impact of the pandemic and work with them to provide those options.With the patient always at the centre of its business and considering the huge development of decentralised clinical trials, the development and implementation of a new service such as Direct-to-Patientseems to be the most natural evolution for CRYOPDP. We have been offering this turnkey solution completely adapted to patients needs, with the same efficiency and total peace of mind, to the benefit of many customers.This model makes life a lot easier for patients and their families, as they dont have to make multiple trips to a clinic or hospital that is potentially a great distance from their home. It is also beneficial for patients who may be too ill to travel, as well as saves patients time and money.Because of these benefits, the direct-to-patient model has increased clinical trial recruitment by up to 60 percent and helped to maintain patient retention by over 95 percent. Drug developers can also gain access to a larger patient population by onboarding those who are not located near participating hospitals or clinics.Patient centricity becomes more evident with the new generation of cell and gene therapies.From day one, CRYOPDP temperature-controlled logistics solutions have always been essential to improve and save patients lives. But, when speaking about cell and gene therapies in particular, this gains an even more significant meaning.The increase in personalised medicine, advanced therapies, and improved access to healthcare in the developing world are influencing future supply chain solutions development. Cell and gene therapies are enabling the healthcare community to shift the arrow and think about patients in a whole new way.Cell and gene therapies demand rigorous and precise temperature control to ensure that the therapies maintain their viability. And maintaining temperature control calls for flawless implementation and execution.From designing the best transportation route to selecting the correct packaging, every detail is critical to keep product integrity high under all conditions, and for this to happen, the healthcare community can count on CRYOPDP specialists who can meticulously handle the entire supply chain process. We've been supporting the life sciences and healthcare communities and focused on improving patient centricity with innovative temperature-controlled logistics solutions because thinking about the patient and the outcome of our work is what moves and inspires us to be better every day.

In all the geographies of the world that we cover, around 150 countries, we produce an operational performance of 99.96%. And to deliver such operational performance, we count on our employees, the specialists around the globe, to follow our quality standards and protocols in detail, so we can deliver a quality service.

When dealing with patients lives, there is no room for errors. Its all about quality of service making sure that lifesaving samples are distributed on time, within the correct specifications and at the right temperature to ensure the patient's health is never compromised.This commitment has helped CRYOPDP to win numerous industry awards, including Best Clinical Trial Logistics Provider in APAC at the Bioprocessing Excellence Awards 2021 and Most Advanced Healthcare Solution Providers from Europe 2021 by Healthcare Insights Magazine.Our achievements are being recognised across the industry, and as we continue to improve our services for healthcare communities, it will be the end patient that benefits the most.

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The Importance of Patient Centricity in Clinical Research and Cell / Gene Therapy Development - Contract Pharma

New York, July 13, 2022 (GLOBE NEWSWIRE) -- According to the World Health Organization (WHO), around 10 million deaths, or nearly 1 in 6 deaths, were caused by cancer in 2020, making it the top cause of death globally. Breast, lung, colon, rectum, and prostate cancers are the most prevalent types of cancer. If found early and appropriately treated, many tumors (30% to 50%) are curable. According to the American Cancer Society (ACS), 1,918,030 new cancer cases and 609,360 cancer deaths are expected in 2022, with lung cancer as the primary cause of death accounting for about 350 of those fatalities daily in the United States.

In recent research titled Global Gene Therapy Market, Kenneth Research provided a brief overview of market elements including growth drivers, restraint factors, current market trends, and potential for future growth. The influence of COVID-19 and its effects on end-users are both thoroughly examined in the market research report, which covers the forecast period, i.e., 2022-2031. In addition, the research study examines the product portfolios and market expansion plans of the principal competitors.In 2020, according to the World Cancer Research Fund (WCRF), there were 18 million new cases of cancer worldwide. 9.3 million of these instances involved men, while 8.8 million involved women. The growth of the global gene therapy market can be attributed on account of the rising prevalence of cancer cases. Also, the adoption of gene therapies for the treatment of cancer is predicted to grow the market further. For instance, at the University of Pennsylvania, the first trial for testing a CRISPR-created cancer medicine was launched in the United States in 2019.CRISPR is a gene-editing-tool, that can modify any DNA segment within the 3 billion letters of the human genome. The global gene therapy market is expected to gather around USD 6 billion in revenue by 2031 and grow with a CAGR of ~34% over the forecast period. Get A Sample Copy of This Report @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented on the basis of region into North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. On the back of rapid rising cancer incidence rates, and the availability of high disposable income, the market in North America is predicted to experience significant expansion over the course of the forecast period. For instance, the Cancer Facts & Figures 2021 by the American Cancer Society, the study estimates that 1.9 million new instances of cancer were diagnosed and 608,570 cancer deaths in the United States in 2021. Also, an increase in the demand for gene-therapy-related R&D activities further helps the growth of the market. According to the World Bank Data, the domestic general government healthcare expenditure in the U.S. was 5,552.60 IN 2019 whereas in Canada the domestic general health care expenditure was 3,873.70 in 2019. Thus, a rise in government health care support is expected to expand the gene therapy-related R&D activities and further aid on to improve the market in the region.On the other hand, the global gene therapy market in the Asia Pacific region is anticipated to experience the greatest CAGR throughout the forecast period owing to the growing population in the region and increased approval and availability of gene therapy products. According to the World Bank data, the total population of China was 1.41 billion in 2020 whereas, India had 1.38 billion people in 2020. As the population grows, the likelihood of contracting a disease increases. Additionally, it is anticipated that increased government efforts to upgrade the health care infrastructure and rising healthcare costs in that region are expected to expand the industry. Also, the health care expenditure in Japan in 2019 was 10.74% whereas, in China, the GDP was 5.35%. In addition to that, the domestic general government health expenditure per capita for Japan was 3,846.54 in 2019 and China was 492.72 in 2019. Thus, growing health care expenditure and government support in health care expansion are further expected for the growth of the market in the region.

Browse to access In-depth research report on Gene Therapy Market with detailed charts and figures: https://www.kennethresearch.com/report-details/gene-therapy-market/10070542

The study further incorporates Y-O-Y Growth, demand & supply and forecasts future opportunities in North America (U.S., Canada), Europe (U.K., Germany, France, Italy, Spain, Hungary, Belgium, Netherlands & Luxembourg, NORDIC[Finland, Sweden, Norway, Denmark], Poland, Turkey, Russia, Rest of Europe), Latin America (Brazil, Mexico, Argentina, Rest of Latin America), Asia Pacific(China, India, Japan, South Korea, Indonesia, Singapore, Malaysia, Australia, New Zealand, Rest of Asia Pacific), Middle East and Africa(Israel, GCC[Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of the Middle East and Africa).The global gene therapy market is segmented by indication into cancer, metabolic disorders, eye disorders, cardiovascular diseases, and others. Among that the cancer segment is predicted to hold the largest share over the forecast period. On account of the growing widespread presence of cancer cases, the growth of the market can be accredited. The estimated number of new cases of cancer patients in India was around 11,57,294 cases which had risen to 13,24,413 total cases in 2020. In addition to that, the total number of cancer patients was 1,708,921 in 2018 in the U.S., according to the Centers for Disease Control and Prevention (CDC) which got increased to an estimated rate of 1.8 million new cases in 2020. The statistical studies exhibit an increasingly widespread of the disease worldwide which is expected to drive the growth of the segment. Gene therapies are used to treat a variety of malignancies, including those of the brain, lung, breast, pancreatic, liver, prostate, bladder, head & neck, skin, and ovary. For instance, according to the World Cancer Research Fund (WCRF), the most common cancers around the world were breast and lung cancers, accounting to 12.5% and 12.2% respectively of all new cases that were expected to be diagnosed in 2020. Also, there were 1.9 million new instances of colorectal cancer, accounting for 10.7% of all cancer cases in 2020.

Get a Sample PDF of Global Gene Therapy Market @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented by end-user into pharma & biotech, and academia. Numerous ongoing researches and studies have been conducted in the pharma and biotech sector which is anticipated to account for the growth of the segment. For instance, based on a study by PhRMA, there were 289 gene therapies done in clinical development by biopharmaceutical companies in 2018 which had increased to 362 gene therapies in 2020. Also, 6 diseases were already being treated using gene therapy, whereas 362 cell and gene therapies were in the development stage in 2020. In addition to that, 9 cell or gene therapy products have been approved by U.S. Food and Drug Administration (FDA) as of February 2020; they are used to treat cancer, eye conditions, and uncommon inherited diseases.

The global gene therapy market is also segmented on the basis of technology and application.

Global Gene Therapy Market, Segmentation by Technology:

Global Gene Therapy Market, Segmentation by Application:

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Some of the well-known leaders in the global gene therapy market that are included in our report are Kineta, Inc., Orchard Therapeutics plc, SIBIONO, Questex, CRISPR Therapeutics, Editas Medicine, and others.

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Global Gene Therapy Market to Garner a Revenue of About USD 6 Billion by 2031 by Growing with a CAGR of ~34% During 2022-2031 - GlobeNewswire