Archive for the ‘Gene Therapy Research’ Category

Gene Therapy in Oncology Market forecast to 2028

The Global Gene Therapy in Oncology Market report provides information about the Global industry, including valuable facts and figures. This research study explores the Global Market in detail such as industry chain structures, raw material suppliers, with manufacturing The Gene Therapy in Oncology Sales market examines the primary segments of the scale of the market. This intelligent study provides historical data from 2015 alongside a forecast from 2021 to 2028.

This report contains a thorough analysis of the pre and post pandemic market scenarios. This report covers all the recent development and changes recorded during the COVID-19 outbreak.

Results of the recent scientific undertakings towards the development of new Gene Therapy in Oncology products have been studied. Nevertheless, the factors affecting the leading industry players to adopt synthetic sourcing of the market products have also been studied in this statistical surveying report. The conclusions provided in this report are of great value for the leading industry players. Every organization partaking in the global production of the Gene Therapy in Oncology market products have been mentioned in this report, in order to study the insights on cost-effective manufacturing methods, competitive landscape, and new avenues for applications.

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Top Key Players of the Market:Bristol-Myers Squibb, Cold Genesys, Advantagene, Amgen, AstraZeneca, Bio-Path Holdings, CRISPR Therapeutics, Editas Medicine, Geron Corp, Idera Pharmaceuticals, Intellia Therapeutics, Johnson & Johnson, Marsala Biotech, Merck, Mologen AG, Oncolytics Biotech, Oncosec, Oncotelic, Shenzhen SiBiono GeneTech, Sillajen Biotherapeutics, Tocagen, UniQure, Ziopharm Oncology

Types covered in this report are: Ex Vivo, In Vivo

Applications covered in this report are: Hospitals, Diagnostics Centers, Research Institutes

With the present market standards revealed, the market research report has also illustrated the latest strategic developments and patterns of the market players in an unbiased manner. The report serves as a presumptive business document that can help the purchasers in the global market plan their next courses towards the position of the markets future.

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Regional Analysis For Gene Therapy in OncologyMarket

North America(the United States, Canada, and Mexico)Europe(Germany, France, UK, Russia, and Italy)Asia-Pacific(China, Japan, Korea, India, and Southeast Asia)South America(Brazil, Argentina, Colombia, etc.)The Middle East and Africa(Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

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In the end, the Gene Therapy in Oncology Market report includes investment come analysis and development trend analysis. The present and future opportunities of the fastest growing international industry segments are coated throughout this report. This report additionally presents product specification, manufacturing method, and product cost structure, and price structure.

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Technical Report on Gene Therapy in Oncology Market 2021 - LionLowdown

These life sciences companies can officially take cash off their Christmas listsafter last weeks bounty.

Certara

Thisbiosimulationcompany took in the second largest life sciences IPO of the year raking in a whopping$668 million. Selling 29.1 million shares at $23 eachput them in at a 19% increase of their original aim.Certara partners with biotech and pharmaceutical companies to accelerate drug development. According to their website, 90% of companies that received new drug approvals by the FDA since 2014 usedCertaras software or services.

AbCellera

AbCellerasdiscovery of the COVID-19 antibody used in Eli Lillysbamlanivimabrocketed this Canadian company into worldwide recognition.Joining the Nasdaq was a natural next step.Themonoclonal antibodywas the first approved by the FDA for the treatment of COVID-19.Originally prepping for a$391 million IPO,AbCelleraupped its offering to 24.15 million shares at $20 per share forexpected proceedsof$483 million, a 24% increase.

Tempus

Mega-raiser Tempus took in another$200 millionin a Series G-2 financing round, bringing their total lifetime raise to $1.05 billion.Currently employing around 1,500, the precision medicine company will use the funds to expand operations and expand to other disease areas includinginfectiousdiseases, depression and cardiology.Tempus AI platform analyzes multi-modal data across major disease types to look for therapeutically relevant insights.

4D Molecular Therapeutics

Offering 1.4 million more shares than planned, 4D raised 23% more, bringing their IPO toraise to$193 million.4D is in a collaboration with pharma giant Roche and has support from Pfizer as well, with these factors adding to the interest of its upsized IPO.The companys target is on both rare and large market diseases, including patient populations that other gene therapies arent able to address.

Locanabio

Building on last years$55 millionraise, San Diego-basedLocanabiosecured$100 millionthis week in a Series B financing round. The funds will be used to advance the companys portfolio of RNA-targeted gene therapies for neurodegenerative, neuromuscular and retinal diseases.Locanabiosunique approach combines gene therapy and RNA modifications to treat disease. Using a gene therapy vector, the treatments deliver RNA-targeting protein tipped with an RNA-modifying enzyme with the potential to treat many diseases linked to the dysfunctional processing of RNA.

Nanobiotix

This French nanoparticle drug developer sold its shares at the low point of their target range, $13.50, but still raked in$99 millionfor their Nasdaq debut.Nanobiotixsproprietary technology, NBTXR3 is a first-in-classradioenhancerto work across solidtumors, enhancing radiotherapy efficacy and producingan immune response with just oneinjection into the tumor. The treatment is currently intwo Phase II studiesfor patients with head and neck cancer.

Reneo Pharmaceuticals

With a focus on genetic mitochondrial diseases, Reneos Series B brought in$95 millionin a financing round led by Novo Ventures andAbingworth.Reneos lead candidate, REN001,has completed an open label safety and tolerability study in patients with primary mitochondrial myopathies. The funds from this raise will take REN001througha Phase II trial.The compoundworks to improve cellular energy metabolism by enhancingmitochondrial function and potentially increasing the number of mitochondria.

Faze Medicines

Biomolecular condensates have been around fordecades, but haverecently begun to gain traction in the biopharma world. Faze is the third company this year to snag investment cash in pursuit of this target. The$81 millionSeriesA will go into the preclinical research in two focus areas:amyotrophic lateral sclerosis (ALS) and myotonic dystrophy type 1 (DM1). The remaining funds will be used to research condensate biology in other disease areas. Faze intends to utilize screening and proteomics techniques to identify proteins that are components of disease-causing condensates.

Rani Therapeutics

This oral biologicscompany brought in$69 millionin a Series E. Looking to transform thehealthcare market, Ranis technology converts injectable drugs into pills. The funds will accelerate the companys internal pipeline of drugs to the clinic and scale up manufacturing."TheRaniPill has the potential to transform major markets where patients must endure frequent and often painful injections," saidMir Imran, Chairman, CEO and founder of Rani Therapeutics. "With this breakthrough platform, capable of creating orally available therapeutic antibodies, peptides, and proteins, we could impact millions of patients worldwide."

Vigil Neuroscience

Launching with a$50 millionSeries A, Vigil is one of many newbiotechssetting out to fight neurodegenerative disease in 2021. The company is developing a pipeline of precision-based therapies to combat both rare and common neurodegenerative diseases by restoring the vigilance of microglia.Atlas cofounded, seeded and incubated Vigil, with pre-clinical stage assets in-licensed fromAmgen Inc.,which will remain a key shareholder.

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Biopharma Money on the Move: December 9-15 - BioSpace

DUBLIN, Dec. 17, 2020 /PRNewswire/ -- The "Gene Expression Market By Product And Services, By Capacity, By Application, And Segment Forecasts To 2027" report has been added to ResearchAndMarkets.com's offering.

Increasing demands for cancer medicines, falling cost of sequencing procedures, and a rise in demand for personalized medicines are key factors contributing to the high CAGR of the gene expression market during the forecast period.

The Global Gene Expression Market is expected to reach USD 6.78 billion by the year 2027, in terms of value at a CAGR of 8.1% over the forecast period. Gene expression promises to tap into a previously unexplored segment in the vast and burgeoning genetic engineering industry.

An increase in investments towards technological advancements and a rise in healthcare expenditure are estimated to shape the growth of the gene expression market. Drug discovery & development and increased demand for personalized medicine in chronic diseases, such as cancer, would be the most lucrative applications for gene expression analysis in the forecast period. Application of gene expression in clinical diagnostics, on the other hand, will reflect a moderate growth throughout the analysis period. Moreover, the falling costs of sequencing have facilitated the integration of genomic sequencing into medicine. With the increased availability and lowering costs of DNA technologies, gene expression has become a more readily used tool indispensable in drug discovery and development. Many companies and educational institutions are collaborating to make gene expression publicly accessible through databases, such as the Connectivity Map (CMap), Library of Integrated Network-based Cellular Signatures (LINCS), and the Tox 21 project.

Further key findings from the report suggest:

Key Topics Covered:

Chapter 1. Market Synopsis

Chapter 2. Executive Summary

Chapter 3. Indicative Metrics

Chapter 4. Gene Expression Market Segmentation & Impact Analysis

Chapter 5. Gene Expression Market By Product and Services Insights & Trends

Chapter 6. Gene Expression Market By Capacity Insights & Trends

Chapter 7. Gene Expression Market By Application Insights & Trends

Chapter 8. Gene Expression Market Regional Outlook

Chapter 9. Competitive Landscape

Chapter 10. Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/im4bgt

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Global Gene Expression Market Analysis and Forecasts - A $6.78 Billion Market by 2027 - PRNewswire

WALPOLE , MA - AUGUST 6: Estelle Lemieux, a 21-month-old with spinal muscular atrophy, practices ... [+] using her new wheelchair outside of her home in Walpole, MA on Aug. 6, 2019. Estelle will be getting a treatment of Zolgensma after her insurer, Aetna, decided to cover the $2.1 million drug. (Photo by Jessica Rinaldi/The Boston Globe via Getty Images)

The promise of gene therapy is to cure diseases associated with faulty or missing genes. Theres enormous potential. Just this month, at the annual American Society of Hematology meeting, it was shown that gene therapy stops bleeding in hemophilia. Researchers reported that a single injection of a viral-mediated gene therapy vector decreases the bleeding rate among patients with Factor IX-related hemophilia B by 91% over a 6 month period.

Ideally, gene therapies address the root causes of disease with a single curative dose. If they can replace a lifetime of expensive maintenance treatments this may lead to cost savings in the long run. Yet, the high upfront costs, uncertainty surrounding long-term durability, and adverse events have led to some concerns among payers and regulators.

Pharmaceutical firms deploy multiple approaches to pursuing curative gene therapy, including:

These approaches build on advances in basic science. Companies involved in gene therapy research and development include mid-size and large firms. Among other large pharmaceutical firms, Bayer is establishing a cell and gene therapy platform within its pharmaceuticals division. The company aims to deploy the platform in as many indications as possible.

Novel drug development is invariably a risky venture. The issue of risk is further amplified in gene therapy. Promising therapies face unexpected challenges. For example, in a surprise decision this fall, the Food and Drug Administration (FDA) rejected BioMarins license application for its gene therapy to treat severe hemophilia A. According to the FDA, valoctocogene roxaparvovec gene therapy, is not ready for approval in its present form. The FDA changed its data requirements for the application. The agency is now requesting that the sponsor BioMarin provide two years of data from the companys ongoing Phase 3 study of the therapy.

While development challenges will persist, payment hurdles may be equally difficult to overcome. Should many of the gene therapies in the pipeline be approved in the coming decade the budgetary impact burden on payers could become overwhelming. Payer concerns stem in part from there being hundreds of gene therapies in clinical development,across a wide range of therapeutic categories, including among others, cardiovascular disease, Parkinsons, various inherited disorders, different types of cancer, viruses such as HIV, and blood diseases like sickle cell anemia.

The churn or turnover at U.S. insurers - as beneficiaries frequently switch plans - lowers the potential return on investment for payers. So, being saddled with high upfront costs without necessarily experiencing the downstream long-term benefits of gene therapies is a considerable problem for which a structural solution has not yet been found.

The payer assumes all the risk with fixed, static pricing. And, the payer isnt able to properly assess that risk, given that clinical development of gene therapies has, thus far, mostly included only very small numbers of patients. Therefore, the real-world benefits and risks remain unclear at the time of approval. Clearly, given the uncertainties regarding long-term durability of gene therapies as well as the potential for toxicity and other adverse effects to patients, a dynamic pricing structure is not only desirable but essentially required for these treatments.

Value-based contracts

In what appear to be harbingers of new ways to finance gene therapies and potentially turn fortunes around of therapies lagging in uptake, drug manufacturers are offering - and in some cases payers have been amenable to - indication-specific pricing arrangements, value-based contracts, and installment plans.

For example, in 2018, the FDA approved the gene therapy Luxturna. This treatment holds the promise to restore functional vision to the blind. The sponsor, Spark Therapeutics, set its products price at $425,000 per eye. Harvard Pilgrim entered into a unique outcomes-based contract with Spark Therapeutics. In the deal, Harvard Pilgrim pays for Luxturna, but gets certain refunds if the treatment wears off after a defined period of time. The full details of the contract are confidential. What is known, however, is that because of federal regulations, known as Medicaid best price rules, the maximum refund cannot exceed 23.1%, or the amount Spark Therapeutics is required to offer Medicaid programs. Spark Therapeutics did request that the Centers for Medicare and Medicaid Services (CMS) offer ways to work around the Medicaid best price requirement, in order for it to be able to accept installment payments and provide insurers deeper refunds or rebates in case the product doesnt meet certain targets.

Novartis Gene Therapies has been working closely with payers to create five-year outcomes-based agreements and novel pay-over-time options for the Zolgensma therapy, indicated for spinal muscular atrophy. The sponsor asserts that the treatment is cost-effective, even when priced at $2.125 million per patient. The installment plans would spread out payments over five years. In addition, the sponsor would offer a refund if a patient dies or the treatment otherwise fails within that period.The current alternative to Zolgensma is BiogensSpinraza, which patients take for the duration of their lifetime. The costs of Spinraza are approximately $4 million over a 10-year span.

In 2019, BluebirdBio told investors it was seeking value-based installment plan contracts to reimburse its sickle cell anemia product LentiGlobin for transfusion-dependent beta-thalassemia. The installments would be paid over a period of up to five years.

After an initial charge, Bluebird Bio would only get reimbursed if the one-time infusion benefits patients. This implies that up to 80% of the cost of LentiGlobin would only be made if there is treatment success. And this success would then be measured and tracked in patient registries maintained by payers.

As part of its contracting preparations, Bluebird Bio has sought ways to bypass Medicaid best price rules; for example, waivers to establish an exemption. The company has also pursued a resolution to the issue of portability - when patients change insurers - by way of a mutual recognition strategy across payers.

But, now the FDA wants Bluebird Bio to provide additional information on the manufacturing process it will use as it transitions the product, LentiGlobin, from clinical trials to commercial production. This will push back the timing of execution of contracts until LentiGlobin gets approved by FDA, which may not be until 2022 or later.

Across the various contract constructs described, payments can be administered in different ways that are not mutually exclusive:

Reimbursement of pharmaceutical products generally happens on a per-unit basis, which spreads out costs over years. But, the cost of a gene therapy is much more concentrated, possibly all upfront in a single payment. Such high upfront one-time costs make it harder for payers to underwrite the risk of full payment for one therapy, let alone the entire range of gene therapies that may be coming to market shortly. Therefore, a combination of installment plans and value-based contracting arrangements will likely be the wave of the future for gene therapy reimbursement.

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Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds - Forbes

Eli Lilly has stepped into the gene therapy space after announcing a deal to acquire Prevail Therapeutics, a company focused on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative diseases.

Lilly will acquire Prevail for $22.50 per share in cash, plus one $4 contingent value right dependent on the first regulatory approval of a product from Prevails pipeline.

This reflects a potential consideration of up to $26.50 per share in cash for a total consideration of approximately $1.04bn.

For Lilly, the acquisition will extend its focus into developing gene therapies, establishing an in-house gene therapy programme anchored by Prevails current portfolio and AAV-based technology.

Prevails pipeline spans clinical-stage and preclinical neuroscience assets, including lead gene therapies PR001 for patients with Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

The companys preclinical pipeline also includes PR004, a potential gene therapy for patients with specific synucleinopathies, as well as candidates for Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders.

"The acquisition of Prevail will bring critical technology and highly skilled teams to complement our existing expertise at Lilly, as we build a new gene therapy programme anchored by well-researched assets, said Mark Mintun, vice president of pain and neurodegeneration research at Lilly.

We look forward to completing the proposed acquisition and working with Prevail to advance their ground-breaking work through clinical development, he added.

For Prevail to achieve the full value of the contingent CVR payment, the first regulatory approval arising from its current gene therapy pipeline must happen by 31 December 2024.

Failing regulatory approval by this date, Lilly said in a statement that the value of the CVR will decrease by approximately 8.3 cents per month until the expiration date 1 December 2028.

Within Prevails clinical pipeline, PR001 has already scored a US Food and Drug Administration (FDA) fast-track designation for the treatment of PD-GBA patients and nGD.

It has also been granted an FDA orphan drug designation for the treatment of Gaucher disease, and rare paediatric disease designation for the treatment of nGD.

Prevails PR006 gene therapy also has an FDA and European Commission orphan designation for the treatment of FTD, with the FDA also handing it a fast-track designation for FTD-GRN.

In November, Lilly signed a deal with Precision BioSciences focused on genome editing research, with an initial focus on developing in vivo therapies for Duchenne muscular dystrophy and two other undisclosed gene targets.

Read more:
Lilly scores gene therapy programme in $1bn Prevail Therapeutics acquisition deal - PMLiVE

I

n a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.

The paper has very strong clinical implications that a single injection maybe is enough for bilateral effects, says Thomas Corydon, who studies ocular gene therapy at Aarhaus University in Denmark and was not involved in the work.

The onset of Leber hereditary optic neuropathy (LHON) is sudden. Patientsusually young menstart losing vision at the center of one eye. Within months, the other eye follows, leaving them legally blind. The disease is caused by a point mutation in the mitochondrial genome that leads to dysfunction and death of retinal ganglion cells, the axons of which make up the optic nerve. About 70 percent of patients have the same mutation, known as MT-ND4.

If you're going to start somewhere, it makes sense to tackle this variant, says Patrick Yu-Wai-Man, an ophthalmologist at the University of Cambridge in the United Kingdom. He and his collaborators, including teams from GenSight Biologics and a group led by University of Pittsburg Medical Center ophthalmologist Jos-Alain Sahel, as well as other groups, previously showed that the point mutation could be corrected in animal models and in cell culture using gene therapy.

Its difficult to get genetic material into the mitochondrial genome because mitochondria have two membranes, an outer and inner membrane, Yu-Wai-Man explains. In the clinical trial, he, Sahel, and colleagues overcame this hurdle by injecting an AAV vector containing a wildtype copy of the ND4gene with an added mitochondrial-targeting sequencea strategy that had already been shown to correctly direct the protein product of ND4 and other mitochondrial genes to the organelle.

Each of 37 patients received the therapeutic virus via a single injection into the vitreous fluid within one eye six to 12 months after the onset of vision loss. They also got a sham treatment in the other eye: a surgeon pressed the eye with a blunt cannula to simulate an injection.

We thought that, if there was going to be an effect, it would be isolated to that eye and then the other one would be the perfect internal control, Yu-Wai-Man tells The Scientist. But as it turns out, that wasnt the case.

With a slight delay in the sham-treated eye, both eyes started to improve. By 96 weeks after treatment, 29 of the patients had gained visual acuity in both eyes and reported increases in their quality of life.

Patients do improve, but, even with the treatment, they still function at a very low level, says Byron Lam, an ophthalmologist at the University of Miami who was not involved in the study. Most of the subjects were still near legal blindness at the end of the study.

To determine how the bilateral effect might be happening, Yu-Wai-Man and colleagues injected the therapeutic virus into one eye of three monkeys. Three months later, they found viral DNA in the noninjected eye and optic nerve. This raises the possibility that the viral vector supplies the wildtype protein in the untreated eye, but its not firm proof.

Finding viral DNA in the untreated eye in primates is a little short of being definitive because DNA expression alone doesnt prove that youre getting a therapeutic effect. Detecting DNA doesnt mean there is mRNA expression or protein production, says Mark Pennesi, an ophthalmologist at Oregon Health & Science University who did not participate in the work.

Previous work has shown that there could be transneuronal spread of the vector, but we also need to keep a critical mind and think that there might be other explanations, agrees Yu-Wai-Man. It could be that injecting the vector in one eye leads to some form of localized inflammation that induces mitochondrial biogenesis, thus making the mitochondria work better, he adds. Another option is that improvement in one eye leads to reorganization in the part of the brain that interprets signals from the eye, which could enhance vision overall.

Clearly, further investigations are needed to understand the underlying mechanisms of how the interocular diffusion of viral DNA vector occurs and whether there are other mechanisms by which the optic nerves directly communicate, Bin Li, an ophthalmologist at Tongji Hospital in China who was not involved in the study, writes in an email to The Scientist.Li explains that his group has also reported that material injected in one eye can reach the other optic nerve.

These findings have implications for how this type of research should be performed in the future, he writes. Theyve shown that contralateral sham-treated eyes cannot serve as true internal control for clinical studies.

When you read this paper, you get a little excited, and then in some ways, you get a little disappointed, because it does look like theres some kind of positive effect with this treatmentthat it does do something more than what would happen with just the natural history of the disease. Unfortunately, the results are confounded by the fact that you treat one eye, but then there is improvement in the untreated control eye, Pennisi tells The Scientist. The question then really becomes . . . why did you get that result?

Along with academic collaborators, Yu-Wai-Man, who consults for GenSight Biologics, will continue to explore this question as they focus on ongoing clinical trials of this therapeutic.

P. Yu-Wai-Man et al., Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy,Science Translational Medicine,doi:10.1126/scitranslmed.aaz7423, 2020.

Correction (December 14): The story has been updated to remove mention of a company that was not involved in the work and to specify which fluid compartment in the eye was injected.The Scientist regrets the error.

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Gene Therapy in One Eye Improves Vision in Both Eyes - The Scientist

Pune, India, Dec. 14, 2020 (GLOBE NEWSWIRE) -- The report mentions that the Gene Therapy Market size was USD 3.61 billion in 2019 and is projected to reach USD 35.67 billion by 2027, exhibiting a CAGR of 33.6% during the forecast period. The global gene therapy market is set to gain momentum from the rising incidence of different types of cancer. The field of this therapy is undergoing several technological advancements that would help in treating cancer in those patients who are at high risks of getting affected by this disease through genetic mutations. In 2019, the U.S. generated USD 2.16 billion in terms of revenue. The country is expected to dominate throughout the coming years stoked by the increasing usage of advanced gene therapies for the treatment of rare conditions.

KEY INDUSTRY DEVELOPMENTS:

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Increasing Innovations & Research Activities to Boost Growth

The U.S Food and Drug Administration (FDA) stated that it is expecting to receive more than 200 applications of this therapy by the end of 2020. This showcases that the rising number of research studies and innovations in this field would affect the gene therapy market growth positively in the near future. In North America, almost 208 companies are currently operating in this market. In addition to this, the Alliance for Regenerative Medicine declared that as of 2018, approximately 259 potential drug candidates are under Phase I clinical trials across the globe.

However, the outbreak of the COVID-19 pandemic is presently impacting the field of research. According to the director of the Office of Tissues and Advanced Therapy (FDA) named Wilson Brayan, nowadays the officials are prioritizing only those drugs that are associated with coronavirus.

To get to know more about the short-term & long-term impact of COVID-19 on this market, please click here: https://www.fortunebusinessinsights.com/industry-reports/gene-therapy-market-100243

The U.S. to Dominate Owing to Presence of Favorable Policies

In 2019, the U.S. generated USD 2.16 billion in terms of revenue. The country is expected to dominate throughout the coming years stoked by the increasing usage of advanced gene therapies for the treatment of rare conditions.

Besides, the presence of favorable reimbursement policies and guidelines would also help in propelling the market growth here. As this type of treatment is not legal in several developing nations, industry giants are emphasizing on the U.S. for launching their products.

Europe, on the other hand, is anticipated to grow significantly backed by the adoption of unique treatment options. Asia Pacific is set to hold a comparatively lower share on account of the decreasing usage of gene therapy because of its expensive nature.

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List of Key Players operating in Gene Therapy Market:

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Global Gene Therapy Market Segmentations:

By Application

Oncology

Neurology

Others

By Vector Type

Viral

Non-viral

By Distribution Channel

Hospitals

Clinics

Others

By Geography

U.S.

Europe (U.K., Germany, France, Italy, Spain, and Rest of Europe)

Asia-Pacific (Japan, China, and Rest of Asia- Pacific)

Rest of World

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Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -...

Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defectiveSMN1gene1.It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein1.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking.Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa."The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy.The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mre Enfant Soleil, Universit Laval in Qubec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3. Without a functionalSMN1gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90%of cases4.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene1.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular AtrophySMA is the leading cause of genetic infant death2. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities5.This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression6.Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly5. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta7,8.

About Novartis in Gene Therapy and Rare DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult http://www.novartis.ca.

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for thein vivogene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon forin vivodelivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

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Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire

DUBLIN, Dec. 17, 2020 /PRNewswire/ -- The "Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global gene therapy market market.

Major players in the gene therapy market are Novartis AG, Bluebird Bio, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, Voyager Therapeutics, Applied Genetic Technologies Corporation, UniQure N.V., Celgene Corporation, Cellectis S.A. and Sangamo Therapeutics.

The global gene therapy market is expected to decline from $3.22 billion in 2019 to $3.18 billion in 2020 at a compound annual growth rate (CAGR) of -1.30%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The market is then expected to recover and reach $6.84 billion in 2023 at a CAGR of 29.09%.

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

North America was the largest region in the gene therapy market in 2019.

The gene therapy market covered in this report is segmented by gene type into antigen; cytokine; suicide gene; others. It is also segmented by vector into viral vector; non-viral vector; others, by application into oncological disorders; rare diseases; cardiovascular diseases; neurological disorders; infectious diseases; others, and by end users into hospitals; homecare; specialty clinics; others.

In December 2019, Roche, a Switzerland-based company, completed its acquisition of Spark Therapeutics for $4.3 billion. With this deal, Roche is expected to strengthen its presence in the gene therapy segment, support transformational therapies and increase its product portfolio. Spark Therapeutics is a US-based company involved in gene therapy.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. For instance, since January 2020, GlaxoSmithKline, a pharmaceutical company, has been investing in AI to optimize gene therapy and develop off-the-shelf solutions for patients. It is also expected to reduce turnaround time and also the cost of gene therapies.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Size And Growth 3.1. Global Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Gene Therapy Market Segmentation 4.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Gene Therapy Market Regional And Country Analysis 5.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

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Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,...

Passage Bio, Inc., a genetic medicines company focused on therapies for rare, monogenic central nervous system (CNS) disorders, has entered into a long-term lease to support Chemistry, Manufacturing and Controls (CMC) lab operations for the companys gene therapy programs. The new lab, scheduled to open in 2Q21 at the Princeton West Innovation Campus in Hopewell, NJ, will initially focus on state-of-the-art analytical capabilities, clinical assay development and validation, biomarker assay validation and clinical product testing to support both viral vector manufacturing and clinical development.The opening of the CMC lab is part of Passage Bios strategy to expand its internal manufacturing capabilities to support its lead gene therapy programs as they move into the clinic and advance toward commercialization. The CMC lab complements the recent opening of Passage Bios dedicated CGMP manufacturing suite at Catalent. These investments provide the company with the foundation for an integrated manufacturing supply chain with capabilities to advance multiple gene therapy programs to support clinical trials worldwide.The 62,000 sq.-ft. lab space is intended to support analytics, process development, quality control and pilot manufacturing. The 1.2 million-sq.-ft., multi-purpose research and development and biologic/pharmaceutical manufacturing campus also provides Passage Bio with expansion opportunities for additional lab space and CGMP manufacturing operations. Passage Bio plans to add more than 20 new positions in 2021 at the new lab.

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Passage Bio Invests In Gene Therapy Manufacturing R&D Site - Contract Pharma

In a development that could restore sight to thousands of people with an inheritable condition calledLeber's Hereditary Optic Neuropathy (LHON), scientists have been able to improve vision in both eyes in a majority of patients even though only one eye was actually treated.

The treatment is an experimental type of gene therapy, where tailored genetic material is injected to counteract genes that are defective or malfunctioning. With LHON, a m.11778G>A mutation in the MT-ND4 gene is the target.

In a phase 3 clinical trial, 37 patients were treated with a modified viral vector rAAV2/2-ND4 in one eye only, leading to an average vision improvement of 15 letters on the standard ETDRS chart you might have spotted at an optician's clinic.

"We expected vision to improve in the eyes treated with the gene therapy vector only," says neuro-ophthalmologist Patrick Yu-Wai-Man, from the University of Cambridge.

"Rather unexpectedly, both eyes improved for 78 percent of patients in the trial following the same trajectory over two years of follow-up."

The eyes that didn't get the gene therapy were given a sham treatment instead, and while the improvement wasn't as great, it was still substantial. Those in the earlier stages of LHON typically saw a bigger improvement in their vision from the treatment.

LHON is the most common form of mitochondrial blindness transmitted from a mother to her children and attacks the retinal ganglion cells, damaging the optic nerves. Around 1 in 30,000 people are thought to be affected, usually men in their 20s or 30s.

The replacement MT-ND4 gene treatment seems to rescue the retinal ganglion cells from their fate, causing results that can be "life-changing" according to the researchers. Normally less than 20 percent of those affected get their sight back.

"As someone who treats these young patients, I get very frustrated about the lack of effective therapies," says ophthalmologist Jos-Alain Sahel, from the University of Pittsburgh.

"These patients rapidly lose vision in the course of a few weeks to a couple of months. Our study provides a big hope for treating this blinding disease in young adults."

While scientists know what causes the loss of vision, finding a way to stop it has proved difficult. LHON is a good candidate for gene therapy though, because it has a clear starting stage and genetic targets that are relatively straightforward to hit.

What's not clear yet is why and how the gene therapy is spreading from one eye to the other. Follow-up experiments in macaque monkeys, which have vision systems similar to humans, suggested the injected viral vector can spread to other tissue via some means of interocular diffusion, but more research is going to be needed to understand the mechanisms at work.

Gene therapy is now being used to tackle a wide range of diseases and health issues, including those inherited from parents. Many other eye problems are in the sights of researchers too, and advances in one area can quickly help research in another something that the team behind the current study is excited about.

"Our approach isn't just limited to vision restoration," says Sahel. "Other mitochondrial diseases could be treated using the same technology."

The research has been published in Science Translational Medicine.

Link:
Experimental Therapy Injected in One Eye Unexpectedly Improves Vision in The Other - ScienceAlert

When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that theyd get office space as soon as her living room table was no longer big enough to hold company meetings.

We lasted about a year before my living room couldnt take it anymore, she said.

With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.

Gene therapy has generated so much enthusiasm for patients and families with these devastating disorders, but theres still a lot of science and innovation left on the table, McMinn said.

The CEO said Neurogene will split the Series B funds into four buckets, the first of which is advancing multiple gene therapy programs into the clinic. She anticipates filing the first IND in 2021 for CLN5, a rapidly progressive subtype of Batten disease caused by a variant in the CLN5 gene.

The second so-called bucket for the Series B funds will be expanding the companys portfolio, followed by another bucket for augmenting our resources for our novel technology platform, the CEO said. Then comes manufacturing.

Weve got the ability to make virus in-house, and the money from the financing will allow us to take that vector to the next stage and make GMP quality vector for use in dosing and clinical trials, McMinn said.

Because Neurogene manufactures products in-house, the biotech has gotten around the massive gene therapy manufacturing bottleneck, which has Big Pharma and big biotech spending billions on retrofitted plants and gene therapy factories.

The concept of gene therapy is simple: A viral particle is used to deliver a healthy copy of a gene to a patient with a dysfunctional gene. In the case of Neurogenes CLN5 candidate, viral vectors shuttle a payload into the body designed to make the CLN5 gene.

Over the next year, key milestones will be filing our first IND, completing the refurbishment of our GMP manufacturing facility, (and) advancing our programs towards the clinic, McMinn said. After CLN5, the goal is to file one to two INDs a year, she added.

The CEO previously served as an analyst at Piper Jaffray, Cowen and Bank of America Merrill Lynch, and as chief business and strategy officer at Intercept. During her time as an analyst, McMinn said most people would stay away from investing in neurology companies because drugs inevitably fail.

Theres really been nothing, very little innovation in devastating neurological disorders, for quite a long time, she said, adding that she was inspired to jump into R&D by an older brother who is neurologically impaired.

Neurogene attracted a slate of new and old investors, including EcoR1 Capital which led the round, and Redmile Group, Samsara BioCapital, Cormorant Asset Management, BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, Alexandria Venture Investments, and an undisclosed leading healthcare investment fund.

For me, I really want to make a difference, McMinn said, adding later, Im personally driven by developing something that is life-altering for people that really have no other option.

Read the rest here:
After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News

Jos-Alain Sahel was on a rare vacation in Portugal in the spring of 2018 when his phone rang with grim news: The gene therapy he had worked on for a decade, a potential cure for a rare form of blindness, had failed in a pivotal trial.

In the first minute, I was very disappointed, Sahel says. I said, well OK, its not working.

A failed trial in drug development is crushing but not unexpected, a tradeoff of doing business in biology. You examine the full data, go back to the drawing board and either abandon the effort or tweak and try again. Sahel, founder of four companies and the longtime head of the Vision Institute of Paris, was used to the process. But this time, when the full data came, he was bewildered.

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They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News

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. Cell therapy involves the transfer of cells with relevant function to the patient. Some protocols use both gene therapy and cell therapy.

Cell And Gene Therapy Market research is an intelligence report with meticulous efforts undertaken to study the right and valuable information. It provides a clear understanding of the subject matter and has been accumulated by suing primary and secondary research techniques.

The study report presents the market overview as market size, revenue, share, forecast and market drivers. In addition, report offers an in-depth analysis about the product scope and market opportunities and market risks for the participants. The report provides the description about the profile of the top manufacturers of Cell And Gene Therapy Market.

Get the PDF Sample Copy of this report @: https://www.a2zmarketresearch.com/sample?reportId=313733 Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

Top Key Players Profiled in this report:

JW CreaGene

Vericel

Tego Sciences

GC Pharma

MolMed

AnGes

Gilead Sciences

Corestem

AVITA Medical

Dendreon

CO.DON

Medipost

Osiris Therapeutics

Amgen

Biosolution

CollPlant

Japan Tissue Engineering

Organogenesis

Orchard Therapeutics

Stempeutics ResearchGlobal Cell And Gene Therapy Market research report offers: Market definition of the global Cell And Gene Therapy market along with the analysis of different influencing factors like drivers, restraints, and opportunities. Extensive research on the competitive landscape of global Cell And Gene Therapy Identification and analysis of micro and macro factors that are and will effect on the growth of the market. A comprehensive list of key market players operating in the global Cell And Gene Therapy market. Analysis of the different market segments such as type, size, applications, and end-users. It offers a descriptive analysis of demand-supply chaining in the global Cell And Gene Therapy market. Statistical analysis of some significant economics facts Figures, charts, graphs, pictures to describe the market clearly.

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Cell And Gene Therapy market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Cell And Gene Therapy markets trajectory between forecast periods.

Regions Covered in the Global Cell And Gene Therapy Market Report 2021: The Middle East and Africa (GCC Countries and Egypt) North America (the United States, Mexico, and Canada) South America (Brazil etc.) Europe (Turkey, Germany, Russia UK, Italy, France, etc.) Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

Get up to 30% Discount on this Premium Report @: https://www.a2zmarketresearch.com/discount?reportId=313733 The cost analysis of the Global Cell And Gene Therapy Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

Reasons for buying this report: It offers an analysis of changing competitive scenario. For making informed decisions in the businesses, it offers analytical data with strategic planning methodologies. It offers seven-year assessment of Cell And Gene Therapy Market. It helps in understanding the major key product segments. Researchers throw light on the dynamics of the market such as drivers, restraints, trends, and opportunities. It offers regional analysis of Cell And Gene Therapy Market along with business profiles of several stakeholders. It offers massive data about trending factors that will influence the progress of the Cell And Gene Therapy Market.

Table of Contents:Global Cell And Gene Therapy Market Research Report 2021-2027Chapter 1 Cell And Gene Therapy Market OverviewChapter 2 Global Economic Impact on IndustryChapter 3 Global Market Competition by ManufacturersChapter 4 Global Production, Revenue (Value) by RegionChapter 5 Global Supply (Production), Consumption, Export, Import by RegionsChapter 6 Global Production, Revenue (Value), Price Trend by TypeChapter 7 Global Market Analysis by ApplicationChapter 8 Manufacturing Cost AnalysisChapter 9 Industrial Chain, Sourcing Strategy and Downstream BuyersChapter 10 Marketing Strategy Analysis, Distributors/TradersChapter 11 Market Effect Factors AnalysisChapter 12 Global Cell And Gene Therapy Market Forecast

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Cell And Gene Therapy Market Trends, Growth, Size, Analysis and Forecast by 2024 with Top Players: JW CreaGene,Vericel,Tego Sciences,GC...

One of the ways scientists hope to offer better treatments for vision loss is through gene therapy, where carefully selected genetic material is injected into the eyes to address mutations. Researchers have been left surprised by the effectiveness of an experimental form of this treatment, which involved an injection into one eyeball yet improved vision across both.

Gene therapies have the potential to treat all kinds of health conditions, ranging from cancer, to diabetes in dogs, to obesity and damaged spinal cords. One area where we're seeing some really exciting progress is in hereditary vision loss, with studies demonstrating the potential of gene therapy to treat color blindness, progressive retinal diseases and glaucoma, with some recently receiving approval from the FDA.

This latest study was conducted by scientists at the University of Cambridge, the University of Pittsburgh and Paris Institut de la Vision, and focuses on a form of inherited vision loss called Leber hereditary optic neuropathy (LHON). This affects around one in 30,000 people and usually occurs in young folks aged in their 20s and 30s, destroying their retinal ganglion cells and in turn the optic nerve. Once the condition takes hold, vision can deteriorate to the point where the subject is considered legally blind in just a matter of weeks, with recovery occurring in less than 20 percent of cases.

The majority of patients suffer from the same mutation affecting the MT-ND4 gene, so the researchers were hopeful of targeting this mutation as a way of improving treatment outcomes for sufferers of LHON. They trialed their gene therapy as part of a study involving 37 patients who had suffered vision loss in the preceding six to 12 months. This meant injecting a viral vector packed with a modified complementary DNA called rAAV2/2-ND4 into the vitreous cavity at the back of just one eye, with a sham treatment injected into the other eye.

We expected vision to improve in the eyes treated with the gene therapy vector only, says study author Dr Yu-Wai-Man. Rather unexpectedly, both eyes improved for 78 percent of patients in the trial following the same trajectory over two years of follow-up.

To investigate the reasons behind this unexpected outcome, the team studied the gene therapys effects in macaques, which have a similar vision system to humans. This enabled them to analyze the tissues from different parts of the eye to see how the viral vector DNA had spread. This provided evidence of interocular diffusion, with the viral vector DNA turning up in the retina, optic nerve and anterior segment of the untreated eye.

As someone who treats these young patients, I get very frustrated about the lack of effective therapies, says senior investigator Dr Sahel, from the University of Pittsburgh. These patients rapidly lose vision in the course of a few weeks to a couple of months. Our study provides a big hope for treating this blinding disease in young adults.

The research was published in the journal Science Translational Medicine.

Source: University of Cambridge

See the rest here:
Single gene therapy injection surprisingly boosts vision in both eyes - New Atlas

New York, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" - https://www.reportlinker.com/p05996809/?utm_source=GNW

The global cell and gene therapy market is expected to decline from $6.68 billion in 2019 to $6.92 billion in 2020 at a compound annual growth rate (CAGR) of 3.61%. The slow growth is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The entire supply chain has been disrupted, impacting the market negatively. The market is then expected to recover and reach $13.23 billion in 2023 at a CAGR of 24.10%.

The cell and gene therapy market consists of sales of cell and gene therapies by entities (organizations, sole traders and partnerships) that develop cell and gene therapies. Cell therapy refers to the transfer of intact, live cells that are originated from autologous or allogenic sources and gene therapy refers to the introduction, removal, or change in the genome for treating diseases. The market consists of revenue generated by the companies developing cell and gene therapy products by the sales of these products.

North America was the largest region in the cell and gene therapy market in 2019. It is also expected to be the fastest-growing region in the forecast period.

In December 2019, Roche, a Swiss multinational healthcare company, acquired Spark Therapeutics for $4.3 billion. The acquisition supports the commitment of Roche to bring transformational therapies and innovative approaches to people with serious illnesses. Spark Therapeutics will continue to work within the Roche Group as an independent company. Spark Therapeutics, headquartered in Philadelphia, is a fully integrated commercial company involved in the discovery, production, and distribution of gene therapies for genetic disorders including blindness, hemophilia, lysosomal storage, and neurodegenerative diseases.

The cell and gene therapy market covered in this report is segmented by product into cell therapy; gene therapy and by application into oncology; dermatology; musculoskeletal; others.

Limited reimbursements preventing patients from receiving treatments are expected to limit the growth of cell and gene therapy (CGT market. In 2019, Trinity Life Sciences, a life sciences solution provider, researched national and large regional commercial health insurance plans in the US. It found that the confluence of increasing price, patient volume and number of CGTs on the market is likely to change the reimbursement model for CGTs and impact payer budgets by 5-10%. Payers realize that financing needs to be generated for cost management due to the uncertainty surrounding reimbursement of ancillary costs. Limited reimbursements and uncertain insurance plans are preventing patients from receiving high-cost CGT, which is expected to limit market growth.

Chimeric antigen receptor (CAR) T-cell therapy is shaping the cell and gene therapy (CGT) market. (CAR) T-cell therapy is a combination of cell and gene therapy in which T cells are collected from the patients blood and are genetically engineered to produce modified receptors at their surface, known as chimeric antigen receptors (CARs). These modified T cells with special structures (receptors) are reinfused into the patient. Then, the modified receptors of T cell help in targeting the surface antigen of the cancer cell that ultimately results in the killing of tumor cells in patients. In 2020, the US-FDA approved Bristol-Myers Squibbs two CAR-T cell therapies to treat lymphoma and multiple myeloma and is set to be launched. Currently, FDA approved CAR-T cell therapy treatments like Tisagenlecleucel for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in children and Axicabtagene ciloleucel for the treatment of adult patients with relapsed or refractory large B-cell lymphoma.

Steady investment and consolidation in cell and gene therapies contributed to the growth of the cell and gene therapy (CGT) market. After recognizing the potential of the CGT market, 16 out of the 20 largest biopharma companies by revenue, added CGT products to their portfolio. For instance, Merck invested $109 million in viral vector and gene therapy manufacturing in April 2020. Moreover, 12% of industrial clinical pipeline products and at least 16% of preclinical pipeline products consist of CGT. Steady investment and consolidation in CGT production capacity led to an increase in production capacity and also contributed to the growth of the market.Read the full report: https://www.reportlinker.com/p05996809/?utm_source=GNW

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Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire

Pune, Maharashtra, India, December 16 2020 (Wiredrelease) Allied Analytics :Cancer gene therapy is an approach for the cancer treatment, where genetically engineered vectors such as viruses are used to transfer or replace a mutated gene (that is causing cancer) with a heathy gene. This technique can be used for both the prevention and treatment of disease. The most commonly used techniques for the treatment using cancer gene therapy includes oncolytic virotherapy, gene transfer, and gene-induced immunotherapy.

Cancer Gene TherapyMarket: Global Opportunity Analysis and Industry Forecast, 2019-2023, the Global Cancer Gene Therapy Market accounted for $289 million in 2016, and is estimated to reach $2,082 million by 2023, registering a CAGR of 32.4% from 2019 to 2023. North America is the highest contributor in the cancer gene therapy market in 2016; however, Asia-Pacific is expected to witness the highest growth rate.

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The key companies profiled in the report include Adaptimmune, GlaxoSmithKline, Bluebird bio, Inc., Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, and OncoGenex Pharmaceuticals.

The rise in prevalence of cancer, ethical acceptance of gene therapy for cancer treatment, and the advancement in this field drive the market growth. In addition, benefits of cancer gene therapy over conventional cancer therapies, increase in government support, and rise in biotechnological funding that encourage the R&D activities for cancer gene therapy fuel the growth of the cancer gene therapy market. However, high cost associated with the treatment and unwanted immune responses are expected to restrain the market growth.

Among the therapy segment, oncolytic virotherapy segment is the largest contributor toward the market growth and gene transfer therapy segment is anticipated to be a lucrative market due to the positive results for these therapies during the preclinical and clinical trials for the treatment of cancer.

In 2016,North Americaaccounted for maximum contribution to the total revenue generated, owing to the high prevalence rate of cancer, presence of high disposable income, and high funding for R&D activities associated with cancer gene therapy. However,Asia-Pacificis expected to witness the highest CAGR during the analysis period, attributable to rise in incidence rate of cancer, increase in government initiative to improve healthcare infrastructure, and rise in healthcare expenditure.

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Allied Market Research (AMR) is a market research and business-consulting firm of Allied Analytics LLP, based in Portland, Oregon. AMR offers market research reports, business solutions, consulting services, and insights on markets across 11 industry verticals. Adopting extensive research methodologies, AMR is instrumental in helping its clients to make strategic business decisions and achieve sustainable growth in their market domains. We are equipped with skilled analysts and experts, and have a wide experience of working with many Fortune 500 companies and small & medium enterprises.

This content has been published by Allied Analytics company. The WiredRelease News Department was not involved in the creation of this content. For press release service enquiry, please reach us at contact@wiredrelease.com.

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Cancer Gene Therapy Market Global Opportunities and Industry Share Expected Surpass $2082 Million b - PharmiWeb.com

FNF Research (fnfresearch.com) offering a comprehensive analysis on the [2020-2026] Gene Therapy Market Report by Quantitative Research Incorporating Impact Of Economic And Non-economic Aspects where users can benefit from the complete market research report with all the required useful information about this market. This is the latest report, covering the current COVID-19 impact on the market. The rapidly changing market scenario and initial and future assessment of the impact are covered in the report. The report discusses all major market aspects with an expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments.

TheGene Therapy Marketreport entails a comprehensive database on Upcoming market estimation based on historical SWOT data analysis. It enables clients with quantified data for current market perusal. It is a professional and detailed report focusing on primary and secondary drivers, market share, leading segments, and regional analysis. Listed out are key players, major collaborations, merger & acquisitions along with upcoming and trending innovation. Business policies are reviewed from the techno-commercial perspective demonstrating better results. The report contains granular information & analysis pertaining to the Gene Therapy Market size, share, growth, trends, segment, and forecasts from 2020-2026.

According to the research report, " [185+ Pages PDF Report] Global gene therapy market was valued at approximately 1.4 billion in 2019 and is expected to reach a value of around USD 3.4 billion, at a CAGR of around 14.7% between 2019 and 2027. Gene therapy is the kind of experimental method that makes use of genes for treating or preventing disease by inserting foreign genetic material like DNA or RNA into the persons cells. "

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(The sample of this report is readily available on request).

The FREE Sample Report Includes:

(**Note: The sample of this report is updated with COVID-19 impact analysis before delivery.**)

Top Market Players Profiles Covered in This Report:

Advanced Cell & Gene Therapy

Audentes Therapeutics

Benitec Biopharma

Biogen

Blubird Bio Inc.

Bristol-Myers Squibb Company

CHIESI Farmaceutici SPA

Eurofins Scientific

Geneta Science

Genzyme Corporation

Gilead

GlaxoSmithKline PLC

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Key Offerings:

The market research report also offers information on potential investment opportunities, strategic growth market analysis, and probable threats that will adhere to the client to systematically and creatively plan out the business models and strategies. The critical data analysis in the Gene Therapy market report is laid out in an upright way. This means that the information is represented in form of infographics, statistics, and uncomplicated graphs to make it an effortless and time-saving task for the client.

Growth of the overall global trust and corporate service market has also been forecasted for the period 2020-2026, taking into consideration the previous growth patterns, the growth drivers, and the current and future trends.

(**The consulting and implementation services segment to account for the highest market share during the forecast period.**)

The scope of the Gene Therapy Market report has a wide spectrum extending from market scenarios to comparative pricing between major players, cost, and profit of the specified market regions. The numerical data is supported by statistical tools such as SWOT analysis, BCG matrix, SCOT analysis, and PESTLE analysis. The statistics are depicted in a graphical format for a clear picture of facts and figures.

The generated report is strongly based on primary research, interviews with top executives, news sources, and information insiders. Secondary research techniques are utilized for better understanding and clarity for data analysis.

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The report provides a complete view of the Gene Therapy market and encompasses a detailed type of portfolio and strategic developments of key vendors. To know the competitive landscape of the Gene Therapy market, an analysis of Porters five forces model is done. The study cover market attractiveness analysis, in which type, source type, and application segments are specialized based on the market size, growth rate, and attractiveness.

The report study further includes an in-depth analysis of industry players' market shares and provides an overview of leading players' market position in the Gene Therapy sector. Key strategic developments in the Gene Therapy market competitive landscape such as acquisitions & mergers, inaugurations of different products and services, partnerships & joint ventures, MoU agreements, VC & funding activities, R&D activities, and geographic expansion among other noteworthy activities by key players of the Gene Therapy market are appropriately highlighted in the report.

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(**FNF Research provides customization of reports as per your need. This report can be personalized to meet your requirements.**)

Table of Contents

Scope of the Gene Therapy Market Report:

Gene Therapy Market Historic Data (2019-2026):

Gene Therapy Market Forecast (2020-2026):

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Research Coverage:

The Gene Therapy market has been segmented based on offering, technology, end-use application, and end-user. It also provides a detailed view of the market across four main regions: North America, Europe, APAC, and RoW.

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Gene Therapy Market Share 2020, By Product Analysis, Application, End-Use, Regional Outlook, Competitive Strategies & Forecast up to 2026 - The...

Los Angeles, USA, Dec. 17, 2020 (GLOBE NEWSWIRE) -- Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade

Glioblastoma Multiforme Clinical Trial Analysis and Pipeline Assessment report offers an analysis of 120+ companies working for the development of therapies for Glioblastoma Multiforme

DelveInsights Glioblastoma Multiforme (GBM) Pipeline Insight, 2020 report proffers a comprehensive coverage of all the companies under pipeline and growth prospects across Glioblastoma Multiforme pipeline, collaborations and agreements taking place, in-depth commercial assessment, and competitive analysis.

Some of the key highlights of Glioblastoma Multiforme Pipeline Report

Glioblastoma Multiforme pipeline is robust and possesses multiple potential drugs in late and mid-stage developments, which are yet to be launched. 120+ companies are dedicatedly working in advancing the GBM pipeline in the foreseeable future.

Some of the Glioblastoma Multiforme Pipeline Therapies

Regorafenib: Bayer Healthcare

Regorafenib is an orally-administered inhibitor of multiple kinases. Sold under the brand name, Stivarga, Regorafenib is approved for use in patients with hepatocellular carcinoma. The drug is currently under investigation for the treatment of Glioblastoma Multiforme which is mentioned. The molecule is in the phase III stage of clinical development.

CYNK 001: Celularity

CYNK-001 is the only cryopreserved allogeneic, off-the-shelf NK cell therapy being developed from placental hematopoietic stem cells as a potential treatment option for various hematologic cancers and solid tumors. The U.S. Food and Drug Administration (FDA) recently cleared Celularitys Investigational New Drug (IND) Application for CYNK-001 in patients with glioblastoma multiforme (GBM).

Know more about GBM pipeline therapies and product profiles, request @ https://www.delveinsight.com/sample-request/glioblastoma-multiforme-gbm-pipeline-insight

Upcoming Glioblastoma Multiforme Therapies

Scope of Glioblastoma Multiforme Pipeline Therapeutics report

Key Questions regarding Current Glioblastoma Multiforme Treatment Landscape and Emerging Therapies Answered in the report

Table of Contents

Browse Detailed TOC, Emerging Drugs and Key Companies @ Glioblastoma Multiforme Drug Pipeline Landscape

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Glioblastoma Multiforme Pipeline Expected to Offer Myriad of Novel Therapies in the Next Decade - GlobeNewswire

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Graphite Bio, a next-generation gene editing company focused on therapies that harness targeted DNA integration to treat or cure serious diseases, today announced that the U.S. Food & Drug Administration (FDA) has cleared its investigational new drug (IND) application for the experimental gene editing therapy GPH101 to initiate a Phase 1/2 clinical trial in patients with severe sickle cell disease (SCD).

The FDA clearance to advance our first investigational therapy, GPH101, into clinical development is a tremendous milestone enabling us to rapidly advance our targeted DNA integration approach into the clinic and bringing Graphite Bio one step closer to making a difference for patients, said Josh Lehrer, M.Phil., M.D., FACC, chief executive officer at Graphite Bio. We are eager to initiate enrollment for the CEDAR clinical trial in early 2021, which will be a historic milestone as the first experimental treatment designed to correct the mutation that is the underlying cause of sickle cell disease. With targeted gene integration, GPH101 seeks to restore normal hemoglobin expression, which has been the ultimate goal of sickle cell disease treatment for more than 70 years.

CEDAR, a Phase 1/2, multi-center, open-label clinical study is designed to evaluate the safety, preliminary efficacy and pharmacodynamics of GPH101 in adult and adolescent patients with severe SCD. An inherited blood disorder affecting 100,000 Americans and millions of people worldwide, SCD is caused by a single mutation in the globin gene that leads to damaged and misshapen red blood cells resulting in anemia, blood flow blockages, intense pain, increased risk of stroke and organ damage, and reduced life span.

GPH101 is Graphite Bios lead investigational therapy that will be evaluated as a potentially curative therapy for patients suffering from SCD. The investigational therapy harnesses the power of CRISPR and DNAs natural homology-directed repair mechanisms to cut out the mutation in the sickle globin gene and paste in the correct natural (wild-type) DNA sequence, with the aim of curing SCD through directly correcting the underlying disease-causing mutation and leading to the production of completely normal red blood cells.

Graphite Bio recently entered into a definitive license agreement for GPH101 with Stanford University, where the investigational therapys preclinical development was advanced at the Center for Definitive and Curative Medicine. The agreement includes exclusive rights to develop selected therapies employing efficient genome editing in hematopoietic stem cells by targeted DNA integration in hemoglobinopathies and several other indications. The preclinical development for GPH101 was led by Stanford Medicine with support from the California Institute for Regenerative Medicine (CIRM).

Todays announcement represents an important step toward making gene editing by targeted DNA integration a therapeutic reality for patients. Clinical entry for GPH101 represents the culmination of decades of research and scientific investment, said Matthew Porteus, MD, PhD, co-founder of Graphite Bio. The scientific platform behind Graphite Bios pipeline was born out of a passion for improving the treatment paradigm for people with sickle cell disease, and it is my hope that this platform can one day offer a cure for this and many other devastating diseases.

About Graphite Bio

Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The companys targeted DNA integration platform harnesses the natural cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD, and is backed by Versant Ventures and Samsara BioCapital. For more information, please visit http://www.graphitebio.com.

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Graphite Bio Receives IND Clearance to Initiate Clinical Trial for Next-Generation Gene Editing Therapy GPH101 in Sickle Cell Disease - Business Wire

DUBLIN--(BUSINESS WIRE)--The "Global Gene Therapy Market - Analysis By Vector Type, By Application, By Region, By Country (2020 Edition): Market Insights, Covid-19 Impact, Competition and Forecast (2020-2025)" report has been added to ResearchAndMarkets.com's offering.

Global Gene Therapy Market was valued at USD 1221.84 Million in the year 2019.

Escalating number of cases related to various chronic diseases including Cancer, Cardiovascular and Neurological Disorders, Alzheimer's and Sickle Cell Diseases, with companies investing heavily in incorporating advanced technology supported by growing collaboration between bio-pharma companies and research institutes to advance in the field of Gene therapy, has been anticipated to infuse growth in the market for Gene Therapy during the forecast period of 2020-2025.

Under the Vector Type segment, AAV vectors, followed by Retrovirus & Gammaretrovirus and Lentivirus, are anticipated to witness the largest market share owing to growing investment in adeno-associated viral (AAV) vectors to advance research programs against strategically selected cell targets. Increasing prevalence of various diseases across the globe will further accelerate the gene therapy market growth during the coming years.

Among the regions, North America, followed by Europe and Asia Pacific, will experience remarkable market share owing to the presence of various leading global companies including Orchard Therapeutics, Voyager Therapeutics, and Spark Therapeutics. With companies investing in adoption of advanced technology supported by enhanced focus on expanding product pipeline by manufacturers to advance in the field of Gene Therapy will further facilitate the market growth during the forecast period.

Scope of the Report

Key Topics Covered:

1. Research Methodology and Executive Summary

1.1 Research Methodology

1.2 Executive Summary

2. Strategic Recommendations

3. Gene Therapy Market: Product Outlook

4. Global Gene Therapy Market: Sizing and Forecast

4.1 Market Size, By Value, Year 2015-2025

5. Global Gene Therapy Market Segmentation - By Vector Type, and By Application

5.1 Competitive Scenario of Global Gene Therapy Market: By Vector Type

5.1.1 Lentivirus - Market Size and Forecast (2015-2025)

5.1.2 AAV - Market Size and Forecast (2015-2025)

5.1.3 Retrovirus & Gammaretrovirus - Market Size and Forecast (2015-2025)

5.1.4 Others - Market Size and Forecast (2015-2025)

5.2 Competitive Scenario of Global Gene Therapy Market: By Application

5.2.1 Neurological Disorders - Market Size and Forecast (2015-2025)

5.2.2 Cancer - Market Size and Forecast (2015-2025)

5.2.3 Cardiovascular Diseases - Market Size and Forecast (2015-2025)

5.2.4 Others - Market Size and Forecast (2015-2025)

6. Global Gene Therapy Market: Regional Analysis

6.1 Competitive Scenario of Global Gene Therapy Market: By Region

7. North America Gene Therapy Market: An Analysis (2015-2025)

7.1 North America Gene Therapy Market: Size and Forecast (2015-2025), By Value

7.2 North America Gene Therapy Market - Prominent Companies

7.3 Market Segmentation By Vector Type (Lentivirus, AAV, Retrovirus & Gammaretrovirus and Others)

7.4 Market Segmentation By Application (Neurological Disorders, Cancer, Cardiovascular Diseases and Others)

7.5 North America Gene Therapy Market: Country Analysis

7.6 Market Opportunity Chart of North America Gene Therapy Market - By Country, By Value, 2025

7.7 Competitive Scenario of North America Gene Therapy Market : By Country

7.8 United States Gene Therapy Market: Size and Forecast (2015-2025), By Value

7.9 United States Gene Therapy Market Segmentation - By Vector Type, and By Application (2015-2025)

7.10 Canada Gene Therapy Market: Size and Forecast (2015-2025), By Value

7.11 Canada Gene Therapy Market Segmentation - By Vector Type, and By Application (2015-2025)

8. Europe Gene Therapy Market: An Analysis (2015-2025)

9. Asia Pacific Gene Therapy Market: An Analysis (2015-2025)

10. Global Gene Therapy Market Dynamics

10.1 Drivers

10.2 Restraints

10.3 Trends

11. Market Attractiveness

11.1 Market Attractiveness Chart of Global Gene Therapy Market - By Vector Type, 2025

11.2 Market Attractiveness Chart of Global Gene Therapy Market - By Application, 2025

11.3 Market Attractiveness Chart of Global Gene Therapy Market - By Region, 2025

12. Competitive Landscape

12.1 Major Technological Innovations, Mergers & Acquisitions and Role of Manufacturers During COVID-19

12.2 Product Pipeline of Leading Gene Therapy Companies

12.3 Market Share Analysis

13. Company Analysis (Business Description, Financial Analysis, Business Strategy)

13.1 Voyager Therapeutics

13.2 Novartis AG

13.3 Spark Therapeutics Inc.

13.4 MoldMed S.P.A.

13.5 Orchard Therapeutics PLC

13.6 Alnylam Pharmaceuticals Inc.

13.7 AnGes Inc.

13.8 Akcea Therapeutics

13.9 BlueBird Bio Inc.

13.10 Sarepta Therapeutics

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

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Gene Therapy Market Analysis by Vector Type, Application, Region - Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -...

BOSTON--(BUSINESS WIRE)--Decibel Therapeutics, a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, today announced that their clinical product candidate, DB-020, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). DB-020 is an investigational therapy for the prevention of cisplatin-induced hearing loss, a serious and debilitating condition for which there are no currently approved therapies.

Cisplatin remains one of the most effective tools we have to treat solid tumors in many patients. Unfortunately, it is associated with significant and irreversible hearing loss and tinnitus in the majority of patients who receive the drug, said Patrick Cobb, M.D., Medical Director, Oncology Research at St. Vincent Healthcare and an investigator in an ongoing Phase 1b clinical trial with DB-020. I've seen first-hand the adverse impact that cisplatin-induced hearing loss has on my patients lives and Im encouraged by the potential to mitigate what has previously been an unavoidable, permanent consequence for these patients.

Decibel is conducting a Phase 1b randomized, double-blind, placebo-controlled, multicenter clinical trial intended to explore the ability of DB-020 to prevent cisplatin-related ototoxicity, which often includes hearing loss, tinnitus and speech recognition problems. DB-020 is being administered to cancer patients for the prevention of cisplatin-induced damage to the inner ear. In this trial, DB-020 is injected into the patients ear shortly before each cycle of cisplatin. The active ingredient, sodium thiosulfate, then diffuses into the cochlea, where it irreversibly binds to cisplatin and prevents it from destroying the sensitive cells necessary for hearing, while not limiting the beneficial anti-tumor effect of cisplatin as a chemotherapy. The statistical power of the study is increased as patients serve as their own control, with one ear receiving treatment with DB-020 and the other receiving placebo.

Fast Track designation is expected to enable us to advance on our goal to provide a meaningful treatment option that prevents patients from experiencing ototoxic effects of cisplatin without risking interference with its important anti-cancer efficacy. We look forward to working with the FDA as we progress our DB-020 clinical program, said Laurence Reid, Ph.D., Chief Executive Officer of Decibel.

FDAs Fast Track designation is designed to facilitate the development and expedite the review of drugs that are being developed to treat serious conditions and fill an unmet medical need. The purpose of the designation is to bring important new drugs to patients earlier across a wide range of diseases.

About DB-020

DB-020 is an investigational therapy for the prevention of cisplatin-induced hearing loss. Decibel completed a randomized, double-blind, placebo-controlled Phase 1 clinical trial of DB-020 in healthy volunteers, in which DB-020 was well tolerated. An ongoing, Phase 1b randomized, double-blind, placebo-controlled, multicenter clinical trial is assessing safety and efficacy in patients receiving cisplatin chemotherapy. Cisplatin, a commonly used chemotherapy agent, is known to cause hearing loss, tinnitus and speech recognition difficulty. DB-020 comprises a proprietary formulation of sodium thiosulfate, or STS, which has been optimized for delivery to the ear. By locally disabling cisplatin in the cochlea, DB-020 is designed to protect hearing without interfering with cisplatins anti-cancer activity.

About Decibel Therapeutics, Inc.

Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibels pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all. For more information about Decibel Therapeutics, please visit http://www.decibeltx.com or follow @DecibelTx.

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Decibel Therapeutics Announces DB-020 Has Been Granted Fast Track Designation by the U.S. Food and Drug Administration - Business Wire

nitpicker/Shutterstock

Less than two months after life sciences giant Bayer acquired N.C.-based AskBio, a gene therapy company, the healthcare giant launched a cell and gene therapy platform within its pharmaceutical division.

This morning, Bayer said the launch of the new platform is a deeply transformative move for its business. Stefan Oelrich, a member of Bayers Board of Management and president of the companys Pharmaceuticals Division, pointed to the impact cell and gene therapies have made in treating diseases and said the companys goal is to be at the forefront of this revolution in science. The company tapped Wolfram Carius, its current vice president of Pharmaceuticals Product Supply, to head the new program.

The C> field is growing at an unprecedented pace. With the establishment of Bayers own C> Platform our company will propel its presence in this area. This will complement our existing C> pipeline which already includes five advanced assets with at least three investigational new drugs annually for the next years, Oelrich said in a statement.

To boost its cell and gene therapy presence, Bayer said it is strengthening its internal capabilities and will also pursue external strategic collaborations, technology acquisitions and licensing. In October, the company acquired AskBio's AAV-based gene therapy pipeline of treatments and its Pro10 AAV manufacturing process, which has become something of a standard across the industry. The Pro10 AAV process is used by multiple companies, including Pfizer, Takeda and Viralgen Vector Core SA.Bayer now owns AskBios pipeline of treatments for Pompe disease, Parkinsons disease, as well as therapies for neuromuscular, central nervous system, cardiovascular and metabolic diseases.

The addition of AskBio complements Bayers other cell and gene therapy company, BlueRock Therapeutics, which itacquired last year. BlueRock is developing induced pluripotent stem cells (iPSC), with its most advanced program aimed at Parkinsons disease. In addition to the two companies, Bayer also acquired a contract manufacturing organization that specializes in gene therapy. Bayer said it has established a C> pipeline that includes five advanced assets and more than fifteen preclinical candidates.

The new C> Platform will combine multiple functions by providing support across the entire value chain for the research and development of cell and gene therapies, the company said. This includes support in preclinical development, CMC, clinical programs, project management and more. The platform will guide projects form concept through commercial launch. The goal is to build robust platforms with broad application across different therapeutic areas, the company said.

The emerging bio revolution represents a once-in-a-lifetime opportunity and a new era for Bayer, said Carius said in a statement. A dedicated C> Platform is vital to accelerate innovation at its source, and to ensure its translation into tangible therapies for patients who have no time to wait.

The C> Platform will allow its partners to operate autonomously to develop and progress their portfolio and technology. The role of Bayers C> Platform is to serve as a strategic guide to ensure the different parts of the organization complement each other and combine the best in Biotech and Pharma know-how.

The formation of the C> Platform comes one day after Bayer and Blackford Analysis entered into a development and license agreement to establish an artificial intelligence (AI) platform for medical imaging. The platform will enable the integration of AI applications into the medical imaging workflow which can support the complex decision-making process of radiologists and is intended to enhance diagnostic confidence, the companies said.

Also this week, Bayer sold most of its stake in Elanco Animal Health for $1.6 billion to cover legal bills from ongoing litigation over the weedkiller Roundup and its alleged role as a carcinogen. Bayer owned approximately 15.5% of Elanco, but faces nearly $11 billion in potential damages related to Roundup lawsuits. In June, the company proposed a $12 billion agreement to resolve Roundup litigation.

Last week, a federal judge rejected a $650 million settlement claim for pollution related to polychlorinated biphenyl, or PCB, which is used to cool heavy-duty electrical equipment.

Most read today on BioSpace:

Original post:
Bayer Launches Cell and Gene Therapy Platform to Maximize Recent Acquisitions - BioSpace

Inc () is a global biotech group focused on unlocking the potential of personalized therapies and closed processing systems through its cell and gene therapy platform.

The Germantown, Maryland-based companys aim is to provide life-changing treatments at the point-of-care to patients at low cost.

The group's Cell and Gene Therapy Biotech platform has three key elements.The first revolves around point-of-care therapeutics, which consists of a pipeline of licensed cell and gene therapies and scientific knowhow. The second aspect relates to point-of-care technologies, which include a suite of in-licensed technologies engineered to create customized processing systems for affordable therapies.

Finally, the third component rests on a point-of-care network, which is a collaborative, international ecosystem of leading research institutes and hospitals committed to supplying cell and gene therapies at the patient bedside. It is an intricate web of affiliated pre-clinical and clinical-stage biopharmaceutical companies, research institutions and hospitals through which is able to in-license technologies or advance therapy medicinal products (ATMPs) and co-develop them with its partners.

On February 11, 2020, Or completed the sale of subsidiary Masthercell Global Inc, a contract development manufacturing organization (CDMO), to Somerset, New Jersey-based Catalent Pharma Solutions, for around $127 million.

The successful sale has spotlighted Orgenesis boss Vered Caplans considerable leadership skills. She has since been named one of the top 20 inspirational leaders in the field of advanced medicine by The Medicine Maker, which creates an annual Power List of top global drugmakers.

Caplan acquired Masthercell in March 2015 and grew the CDMO segment revenue from a run-rate of $3 million to a run-rate of around $30 million at the end of 2019, reflecting a compound annual growth rate of 59% under her leadership, and a sale price of more than five times the initial purchase price of around $25 million.

Caplan has indicated that she plans to use the Masthercell sale proceeds to grow Orgenesis's evolving point-of-care cell therapy business and develop advanced therapy medicinal products.

In November, Orgenesis posted third-quarter results that saw its revenue jump 40% year-over-year on the back of growth in its point-of-care cell and gene therapy network. For the period ended September 30, 2020, the company reported revenue of $1.7 million, compared to $1.2 million in the same period in 2019.

The company also maintained a solid balance sheet with $88.8 million in cash and equivalents at the end of September, reflecting the successful sale of its Masthercell Global CDMO.

Orgenesis is using the Masthercell sale proceeds to expand the companys point-of-care cell therapy business.

In October, the group completed the acquisition of Koligo Therapeutics Inc, a regenerative medicine company, including all of the assets of Tissue Genesis LLC. The agreed consideration terms include $15 million in shares of Orgenesis stock valued at $7 share which will be issued to Koligos accredited investors (with certain non-accredited investors to be paid solely in cash) and an assumption of $1.3 million in Koligos liabilities, estimated to be nearly all of Koligos liabilities.

Koligos management team will be joining Orgenesis to continue commercial and development activities. Koligo CEO Matthew Lehman is an accomplished executive in the biotech and regenerative medicine fields. Orgenesis believes the acquisition will help to expand its therapeutic and technology resources, while adding a highly experienced US team to help further bolster Orgenesis point-of-care network in the US.

On the therapeutics front, Orgenesis is focused on several key verticals, including cell-based cancer immunotherapies, treatments for metabolic and neurodegenerative diseases and tissue regeneration, as well as antiviral therapies. The groups near-term goal is to expand the availability of KYSLECEL from the recent Koligo acquisition. KYSLECEL, is made from a patient's own pancreatic islets the cells that make insulin to regulate blood sugar and is commercially available in the US for chronic and recurrent acute pancreatitis.

Like many companies, Orgenesis is also focused on the coronavirus (COVID-19) space. It is planning patient recruitment for a phase 2 randomized clinical trial of Koligos KT-PC-301, subject to US Food and Drug Administration (FDA) review and clearance of an investigational new drug (IND) application. KT-PC-301 is an autologous clinical development-stage cell therapy candidate for COVID-19-related Acute Respiratory Distress Syndrome, which Orgenesis acquired as part of the Koligo acquisition.

Koligos KT-PC-301 is a cell therapy that is derived from a patients own adipose (fat) tissue. A small amount of fat is collected from the patient and sent to Koligos manufacturing facility in Indiana to make KT-PC-301. The product is made within hours and sent back to the hospital for intravenous administration. KT-PC-301 contains mesenchymal stem cells, vascular endothelial cells, and immune cells which migrate to the patients lungs and other peripheral sites of inflammation. Published nonclinical and clinical evidence demonstrates that KT-PC-301 may stabilize microcirculation to improve oxygenation; maintain T and B lymphocytes to support antibody production, and induce an anti-inflammatory effect.

In addition, Orgenesis is gearing up for a Phase 2 study of Ranpirnase for the treatment of conditions caused by human papillomavirus, pending a planned IND submission to the FDA.

Meanwhile, Orgenesis has advanced the development of a cell-based vaccine platform targeting COVID-19 and other viral diseases. It has launched BioShield to set up a first line of defense against COVID-19 through the discovery of neutralizing human antibodies to contain the spread of the viral pathogen. In addition, Orgenesis has signed an agreement with Fortune 500 science giant to develop, and potentially obtain FDA approval of Ranpirnase for treating COVID-19 patients. Ranpirnase has shown preclinical antiviral activity in diseases such as influenza, Ebola, SARS, and cytomegalovirus. Orgenesis acquired the broad-spectrum Ranpirnase antiviral as a part of its $12 million cash and stock deal to acquire Tamir Biotechnology.

Orgenesis intends to leverage its network of regional partners to advance the development of its pipeline. Towards this end, its partners have committed to funding the clinical programs. In turn, Orgenesis typically grants its partners geographic rights in exchange for future royalties, and a partnership with Orgenesis to support the supply of the targeted therapies. Caplan has said that through this unique business model, Orgenesis has already signed contracts, which could generate over $40 million in revenue over the next three years, if fully realized.

There are also plans to continue to develop, license and form partnerships around a variety of point-of-care technologies to support work in areas such as tumour-infiltrating lymphocytes, CAR-T, CAR-NK, dendritic cell therapies, and mesenchymal stem cell-based therapies.

Currently, the costs of cell and gene therapies are prohibitive, as illustrated by CAR-T therapies, which cost hundreds of thousands of dollars per patient. To lower costs, Orgenesis is switching from a high-cost centralized manufacturing model to a localized point-of-care model.

In April, Orgenesis teamed up with regenerative medicine and cell therapy firm RevaTis on a joint venture to produce certain stem cells. The two firms plan to leverage Orgenesiss autologous CGT Biotech platform to advance clinical trials. Under the deal, RevaTis and Orgenesis will use the formers patented technique to obtain muscle-derived mesenchymal stem cells as a source of exosomes and other cellular products. The companies are hoping to use Orgenesiss expertise and point-of-care platform, which include automated systems, 3D printing and bioreactor technologies.

Meanwhile, Orgenesis and ExcellaBio have developed a breakthrough manufacturing process for so-called bioxomes, which are synthetically made exosomes or extracellular vesicles (EVs). The latter are what transfer DNA, RNA, and proteins to other cells, thereby altering the function of targeted cells. Until now, exosome/EV production has been based on conventional, complex and costly methods of ultrafiltration. However, the two companies have demonstrated the scale-up of bioxomes through a proprietary technique, while generating consistent and repeatable results, including uniform particles sizes.

Orgenesis has added the University of California (UC), Davis to its point-of-care network. The first project with UC Davis Health is on developing a lentiviral manufacturing system which will address a significant unmet need in the market for an efficient and scalable manufacturing process.

Orgenesis CEO Vered Caplan noted that the company has dramatically transformed in 2020 helped by the Masthercell sale.

The sale was an important step in our strategy to leverage our unique capabilities that directly address the key challenges facing the cell and gene therapy industry, Caplan said in a statement.

Our goal is to transform the delivery of cell and gene therapy through our point-of-care therapeutics, technologies and network, thereby lowering costs and unlocking the power of cell and gene through a more decentralized and integrated approach, she added.

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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