Archive for the ‘Gene Therapy Research’ Category
Solvias Acquires Cergentis to Bolster Biologics and Cell & Gene Therapy Capabilities – PR Newswire
Cergentis is a cornerstone acquisition that expands Solvias' solutions supporting complex and emerging therapies.
According to pharmaceutical market intelligence provider, Evaluate, global sales of cell and gene therapies are projected to accelerate at a 63% compound annual growth rate through 2026. As more researchers leverage emerging genetic engineering techniques to develop complex, novel medicines, they require sophisticated solutions to analyze their safety and effectiveness.
With the addition of Cergentis, Solvias supports the increasing number of global pharmaceutical, biotech, and contract development and manufacturing organizations developing genetically engineered therapies with an expanded platform of testing services highlighted by:
Archie Cullen, CEO, Solvias, stated:"We are relentlessly focused on ensuring the safety of new therapies in development. Cergentis is a cornerstone acquisition that expands our solutions supporting complex and emerging therapies. We will continue to pursue strategic acquisitions that add specialized capabilities to our offering and advance our goal of being a forerunner in our industry."
Joris Schuurmans, CEO, Cergentis, added:"We are excited to become part of a global leader that complements our scientific expertise, innovation and customer service. Solvias and Cergentis share a deep commitment to providing our customers with the highest quality solutions and support to safely get their products into the hands of patients who need them."
Effective immediately, Mr. Schuurmans will join Solvias' leadership team and continue to lead operations for Cergentis.
Cergentis marks Solvias' second acquisition since partnering with health care investors Water Street Healthcare Partners and JLL Partners in 2020. The company has recruited industry leaders to join its board and commenced a program to significantly upgrade and expand its information technology platform and infrastructure to support its plans for global expansion.
Financial terms of the acquisition are not being disclosed. Achelous Partners served as the advisor to Cergentis on the transaction.
About CergentisCergentis is a trusted genomics-focused biotechnology company providing services and in-house solutions based on its proprietary genomic analysis platform to all leading biopharmaceutical companies and renowned research institutes. With widely published and recognized genetic analyses, Cergentis supports a global customer base in the characterization and QC of genetically engineered models, biopharmaceutical cell line development, and cell- and gene therapy products. By helping to de-risk R&D program decisions, minimizing time-to-clinic, providing objective genomic evidence for regulators, and supporting patient safety, Cergentis aims to support biopharmaceutical medicine development programs worldwide. For more information, visit cergentis.com.
About Solvias AGSolvias is a worldwide leader in contract research, development and manufacturing for the pharmaceutical, biotech, material science and cosmetic industries. Drawing on 20 years of scientific excellence, the company provides flexible and scalable analytical and manufacturing solutions that ensure the integrity of pharmaceutical and medical device products across their life cycle. Headquartered in Kaiseraugst near Basel, Switzerland, Solvias and its laboratories operate to the highest standards and in accordance with ISO, GMP, GLP and FDA regulations. For more information, visit solvias.com.
SOURCE Solvias
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Solvias Acquires Cergentis to Bolster Biologics and Cell & Gene Therapy Capabilities - PR Newswire
GenScript ProBio, DAAN Bio Therapeutics Enter Cell and Gene Therapy Partnership – Contract Pharma
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Breaking News
Sign MOU for the discovery and development of novel drugs.
07.14.22
Through this agreement, both companies will cooperate in the overall development process, such as discovery, manufacturing and clinical trials of antibody and cell treatments, and related research and service projects.
DAAN Bio Therapeutics has conducted single cell transcriptomic and proteomic biomarker analysis through its own analysis platform using tissues secured from solid cancer patients along with DAAN Cancer Research Institute and Yonsei New Il Han Institute for Integrative Cancer Research.
Accordingly, DAAN Bio Therapeutics signed a contract manufacturing organization (CMO) service through GenScript ProBio to develop antibodies that specifically bind to the discovered target material, and established its own bispecific antibody production pipeline using antibodies derived through GMP production.
I hope this strategic partnership with DAAN Bio Therapeutics will be of great support as a global partner in accelerating the development of DAAN's antibody and cell therapy through the GenScript ProBio's one-stop CDMO service platform, said Brian Min, CEO, GenScript ProBio.
Byoung Chul Cho, co-founder and CEO, DAAN Bio Therapeutics, said, This strategic cooperation will be an opportunity to revitalize the development of antibody and cell therapy that control unique cancer treatment targets secured by DAAN Bio Therapeutics using GenScript ProBio's qualified and competitive technologies.
Through this partnership, both GenScript ProBio and DAAN Bio Therapeutics will continue their cooperation more closely with each other to develop subsequent pipelines of DAAN Bio Therapeutics, such as antibody drug and cell therapy.
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GenScript ProBio, DAAN Bio Therapeutics Enter Cell and Gene Therapy Partnership - Contract Pharma
BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and…
BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress July 9-12, Including 4 Oral and 2 Poster Presentations
Commitment to Advancing Care for People with Hemophilia A Demonstrated with Largest and Longest, OngoingClinical Development Program for any Gene Therapy in Hemophilia A
European Commission Approval for Valoctocogene Roxaparvovec Expected Q3 2022
SAN RAFAEL, Calif., July 11, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that it presented four oral presentations and two poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 World Congress from July 9-12 in London.
"With each passing year, the breadth of data supporting valoctocogene roxaparvovec increasingly demonstrate the positive impact gene therapy treatment may provide people with severe hemophilia A. These presentations at ISTH offer supportive evidence of long-term hemostatic efficacy, consistent safety results in clinical studies, efficacy from our pivotal study that is consistent with propensity scoring, the clearance of the vector, and on health-related quality of life over six years," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We look forward to an anticipated approval of valoctocogene roxaparvovec in Europe and providing a therapy that could represent an important and valuable treatment choice for patients with severe Hemophilia A by offering the potential to reduce both the burden of the disease as well as the burden of treatment."
"BioMarin continues to increase and share important data about investigational valoctocogene roxaparvovec that may be useful for patients and physicians to evaluate therapeutic options based on an individual's unique circumstances," said one of the presenters Professor Johnny Mahlangu, a study investigator and Professor in Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand in Johannesburg, South Africa.
Presentation of these data at ISTH follows the Committee for Medicinal Products for Human Use (CHMP) adopting a positive opinion recommending conditional marketing authorization (CMA) for valoctocogene roxaparvovec for adults with severe hemophilia A. Valoctocogene roxaparvovec is the first gene therapy to be recommended for approval in Europe for hemophilia A. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.
BioMarin's presentations at ISTH include:
Oral Presentations
Comparative effectiveness of valoctocogene roxaparvovec and prophylactic factor VIII replacement estimated through propensity scoring
Anthony J. Hatswell, PhD, Director and Analyst, Delta Hat Limited, Nottingham, UK
Sunday July 10, 2022, 3:45 PM - 4:00 PM BST
Relationship between transgene-produced FVIII and bleeding rates 2 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1
Professor Johnny Mahlangu, Professor of Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand in Johannesburg, South Africa.
Sunday July 10, 2022, 3:15 PM - 3:30 PM BST
Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A
Professor Michael Laffan, faculty of Medicine, Department of Immunology and Inflammation at Imperial College London, Director of the Hammersmith Hospital Haemophilia Centre
Sunday July 10, 2022, 3:00 PM - 3:15 PM BST
Innate and Adaptive Immune Responses to Adeno-associated viral Gene Therapy in the Severe Hemophilia A Dog Model
Paul Batty, MBBS, PhD, Associate Professor at University College London and an Honorary Consultant in Haemostasis and Thrombosis at the Katharine Dormandy Haemophilia and Thrombosis Centre (Royal Free Hospital, London, UK)
Saturday, July 9, 2022, 2:00 PM 2:15 PM BST
Posters
Comparability of bleeding outcomes by prophylactic FVIII replacement intensity: A post hoc analysis of a noninterventional study of men with severe hemophilia A
Charlotte Camp, MSc, Associate Director, Health Economics and Outcomes Research, BioMarin
Monday July 11, 2022, 6:30 PM - 7:30 PM BST
Blood Biodistribution and Vector Shedding of Valoctocogene Roxaparvovec in People with Severe Hemophilia A: Results from the Phase 3 GENEr8-1 Trial
Suresh Agarwal, PhD, MS, RPh, Director, Clinical Pharmacology, BioMarin
Sunday July 10, 2022, 6:30 PM - 7:30 PM
About Hemophilia A
People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed. The standard of care for individuals with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.
Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of seven commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visitwww.biomarin.com.
Forward-Looking Statements
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements about: the data presented at ISTH, including the four oral and two poster presentations, the development of BioMarin's valoctocogene roxaparvovec program generally, the impact of valoctocogene roxaparvovec gene therapy for treating patients with severe hemophilia A, a final approval decision for valoctocogene roxaparvovec, typically consistent with the CHMP recommendation, expected from the European Commission in Q3 2022 and the potential to transform the lives of these patients and the ongoing clinical programs generally. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec, including final analysis of the data from these trials and the entire development program, including further assessment of safety events, any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA and other regulatory authorities; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.
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BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and...
Gene Editing Market is Expected to Reach USD 13.77 Billion With CAGR of 18% By Forecast 2027 Says Maximize Market Research (MMR) – Digital Journal
Gene Editing Market Is Expected To Reach USD 13.77 Billion By 2027 At A CAGR Of 18 percent.
Maximize Market Research has published a report on theGene Editing Marketthat provides a detailed analysis for the forecast period of 2022 to 2027.
Gene Editing Market Scope:
The report provides comprehensive market insights for industry stakeholders, including an explanation of complicated market data in simple language, the industrys history and present situation, as well as expected market size and trends. The research investigates all industry categories, with an emphasis on key companies such as market leaders, followers, and new entrants. The paper includes a full PESTLE analysis for each country. A thorough picture of the competitive landscape of major competitors in the Gene Editing market by goods and services, revenue, financial situation, portfolio, growth plans, and geographical presence makes the study an investors guide.
Request Free Sample:@https://www.maximizemarketresearch.com/request-sample/2748
Gene Editing Market Overview:
The prospect of treating genetic abnormalities, infectious illnesses, and cancer via gene editing is promising. Gene editing is a form of genetic modification in which, with the use of a designed nuclease or molecular scissor, DNA is added to, removed from, or replaced in an organisms genome in order to treat a specific condition. These nucleases damage DNA at specified sites in the genome to produce double-strand breaks. Targeted mutations result from the repair of the generated double-stranded breaks by non-homologous end joining or homologous recombination (edits).
Gene Editing Market Dynamics:
Gene-editing technologies ongoing technological breakthroughs are a key element fueling the markets growth. Additionally, government financing is readily available, there is more genomics research underway, and cancer and other genetic illnesses are becoming more common, all of which contribute to the markets growth. The rise of the genome editing market is also aided by the creation of breakthrough diagnostic tools based on CRISPR that help to lessen the negative effects of the COVID-19 pandemic.
However, there are several constraints that limit the markets growth to some extent, including the high cost of genomic equipment and rising worries about the dangers and moral dilemmas related to genome editing. However, the presence of major players in developing nations and applications in various stages of the drug development process are a few variables that soon open up profitable potential for the genome editing industry.
The gene editing market appears to be fragmented because there are numerous major and minor significant players. These market participants look to gain a competitive edge through strategic actions like partnerships, acquisitions, growths, collaborations, and the introduction of new products and technologies. Growing investments in genomics R&D, the prevalence of hereditary diseases and chronic disorders, and rapid advancements in genetic engineering are key factors supporting the market share for gene editing.
The market for genome alteration is hampered by certain problems. It is expected that ethical issues with genetic editing, such as safety and the high cost of gene therapies, may somewhat restrain market growth over the predicted years. Market limitations also are brought about by rigorous regulatory rules and unfavourable public opinions of genetic research.
The leading companies in the gene editing industry may face difficulties due to a number of market-related worries. This includes the price of the available genetic editing tools. The markets potential growth may be constrained by expensive maintenance and treatment expenses for genome alteration therapies and associated treatments, as well as by a lack of infrastructure in medium- and low-income countries.
Gene Editing MarketRegional Insights:
During the forecast period, North America is expected to hold a sizable portion of the worldwide genome editing market. The market in the region is expected to expand in the coming years due to the development of gene therapy in the United States, an increase in the consumption of genetically modified crops, an increase in the prevalence of infectious diseases and cancer, and the accessibility of research grants and funding.
Gene Editing MarketSegmentation:
By Technology:
By Application:
By End User:
Gene Editing Market Key Competitors:
To Get A Copy Of The Sample of the Gene Editing Market, Click Here:@https://www.maximizemarketresearch.com/market-report/gene-editing-market/2748/
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Gene Editing Market is Expected to Reach USD 13.77 Billion With CAGR of 18% By Forecast 2027 Says Maximize Market Research (MMR) - Digital Journal
Automating the Genomic Medicines of the Future – Bio-IT World
Contributed Commentary by Per Hammer, Cytiva
July 15, 2022 | Historically, the heavily regulated biopharma industry has been slow to adopt new technologies. However, a shift toward automation is vital to ensure that next-generation solutionssuch as cell and gene therapiesare produced at scale.
Less than one in five senior pharma executives strongly believe that frontier technologies, such as artificial intelligence, are widely adopted to support automation and increase the speed of specific processes. With cell therapies approved by global regulatory bodies, it is time to accelerate smart technologies and cell and gene therapy manufacturing.
Todays cell therapy treatments are often made on a small-scale, include manual preparation steps, and are produced for a clinical trial setting. Researchers spend days processing cellular material, monitoring its growth during the expansion phase, and preparing for re-administration to the patient. This process is demonstrated in administering autologous treatments so that every patient receives a unique living drug.
Though the current process is complex, it offers inspiring outcomes. For example, on April 1, 2022, the Food and Drug Administration (FDA) approved Kite Pharmas Yescarta, a chimeric antigen receptor (CAR) T-cell therapy for adult patients with large B-cell lymphoma. This kind of cancer is usually resistant to initial treatment and relapses within one year. With FDA approval, Yescarta (axicabtagene ciloleucel) is now the second-line treatment, a first for an autologous CAR T-cell therapy.
Cell Therapy Enters Mainstream
The exceptional results emerging from cell therapy clinical trials suggest we are entering a new phase of medical treatmentone where we can expect far more from our healthcare interventions than we ever imagined. Following the regulatory approval of autologous CAR T-cell therapies, the global cancer treatment landscape is changing, and the future is bright.
The success of COVID-19 vaccines signaled the arrival of the genomic medicines ageone where we hope to see cell and gene therapies deliver long-term remission and even cures for patients with some of the most complex diseases. According to the Alliance for Regenerative Medicine 2021 Annual Report, nearly 60% of the ongoing regenerative medicine clinical trials studied prevalent diseases by the end of the calendar year. But to get these powerful treatments to those who need them, we must have an automated manufacturing infrastructure that can generate cell therapies to meet increased demand in the coming years.
Saving Time Through Automation
Time is of the utmost importance, as biopharma manufacturing involves patient cells that have limited viability. Manual approaches to cell therapy production are time-consuming, and tasks such as checking cells at regular intervals during expansion are laborious. Another time-draining factor is the workflow and cleaning routines involved in maintaining a safe lab environment.
Automated solutions reduce or remove many of these challenges. After setting up a process, an operator can focus on other things while critical parameters such as temperature, pH level, gas transfer, and flow rates are monitored and controlled without human intervention.
Reducing Risks for Better Results
Manual cell processing solutions are complex, with many checkpoints across isolation, expansion, harvesting, and preservation stages. Unfortunately, each of these steps increases the risk potential. Despite the research teams expertise, there is still a chance that materials could be inadvertently contaminated during numerous open stages.
Additionally, limited process control can lead to difficulties in achieving high reproducibility. An automated modular solution minimizes these risks by bringing multiple steps within a closed, highly regulated, and controlled system.
Improving Manufacturing Efficiency
Changing a manufacturing process requires multiple manual routines and adjustments that must be checked and documented. However, documentation and protocols are less helpful when a customized process is used because they only apply to that specific setup.
Standardization would effectively improve manufacturing efficiency. This approach would ensure that what is learned in one project can be referenced in future work, with data and documentation applicable across different technology applications. A modular chain of connected systems allows for process variation with instruments running in customized configurations. Additionally, having control of an individual instrument leads to the straightforward use of built-in software and sensors.
Automated Manufacturing: The Way Forward for Cell and Gene Therapy
By using automated manufacturing to minimize human interaction, time, and resource requirements, it is possible to increase production speed and lower some risks and costs associated with commercialization.
The industry is ever-changing and adjusting its complex, yet exciting challenges will take some time. However, automation can create a significant advantage over competitors, providing the tools needed to produce cell therapies with the highest levels of safety and efficacy for patients.
Per Hammer has two decades of experience in the biopharma industry, mainly supporting customers in academics through process development and manufacturing. Per joined Cytiva in 2001, taking on several distinct roles in the company. Most recently, he progressed from Product Manager Leader for the Bioprocess Automation and Digital Team to Senior Global Product Manager for the Cell & Gene Therapy Automation and Digital Solutions. He can be reached at per.hammer@cytiva.com.
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Automating the Genomic Medicines of the Future - Bio-IT World
Adverum Biotechnologies Presents Best-Corrected Visual Acuity and Central Subfield Thickness Analyses After a Single IVT Injection of ADVM-022…
- Study participants had an 81%-98% reduction in annualized anti-VEGF injections and demonstrated continuous aflibercept expression levels through three years
- Mean best-corrected visual acuity and central subfield thickness were maintained or improved in subjects treated with ADVM-022
-The Phase 2 LUNA trial is expected to dose the first subject in the third quarter of 2022 and preliminary data anticipated throughout 2023
REDWOOD CITY, Calif., July 15, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced new data from the OPTIC study treating wet age-related macular degeneration (wet AMD) during the American Society of Retina Specialists (ASRS) 2022 Annual Meeting. New data presented are as of February 24, 2022 and include best-corrected visual acuity (BCVA) and central subfield thickness (CST) maintenance, as well as reduction in CST fluctuation after a single, in-office intravitreal (IVT) injection of ADVM-022, ixoberogene soroparvovec (Ixo-vec), in subjects requiring frequent anti-VEGF injections for their wet AMD.
We are pleased to present our findings on BCVA and CST from the OPTIC trial, establishing that maintenance in both BCVA and CST, as well as a reduction in CST fluctuations, were sustained through at least two years, suggesting the long-term durability of Ixo-vec after a single IVT injection, stated Richard Beckman, M.D., chief medical officer at Adverum Biotechnologies. As several publications have recently highlighted, retinal CST fluctuations over time are associated with poor long-term visual outcomes for patients. The combination of durable maintenance of BCVA and reduced CST fluctuations in subjects who previously required frequent IVT injections further support our belief that Ixo-vec can provide better long-term benefit for wet AMD patients. We are excited by the possibility of extending the treatment benefit for this lifelong disease from the order of months to the order of years.
Data Highlights as of February 24, 2022
We are excited to present these encouraging data, showing continuous and stable aflibercept expression for over three years in our OPTIC trial in subjects with wet AMD, commented Laurent Fischer, M.D., president and chief executive officer at Adverum Biotechnologies. As we recently announced, we completed our IND amendment with the U.S. Food and Drug Administration, received European Medicines Agency PRIME designation, and are preparing to initiate the Phase 2 LUNA trial of Ixo-vec in wet AMD. The LUNA trial was designed after a detailed data review from all 55 participants treated to date with a single ADVM-022 injection. Four new enhanced prophylactic steroid regimens will be evaluated in LUNA with the aim of providing steroid coverage during the period of peak immunogenicity. Our goal is to enhance the safety profile of ADVM-022 while building upon the impressive efficacy profile we continue to see in the OPTIC trial. We plan to dose the first subjects in the third quarter of 2022 and anticipate preliminary data from LUNA throughout 2023 and look forward to providing more detail on the expected timeline once we can assess the pace of enrollment.
The LUNA trial is a multicenter, double-masked, randomized, parallel-group Phase 2 trial evaluating two doses of Ixo-vec, including 2E11 and a new, lower 6x10^10 vg/eye (6E10) dose, in up to 72 patients with wet AMD. The LUNA trial will assess four new enhanced prophylactic steroid regimens, including local steroids and combinations of local and systemic steroids to test the relative contribution of local versus systemic AAV exposure on ocular inflammation. Specific regimens include topical difluprednate (Durezol), IVT Ozurdex, or a combination of either topical Durezol or IVT Ozurdex with oral prednisone.
The trial will randomize the participants equally between the 2E11 and 6E10 Ixo-vec doses across four prophylactic steroid regimens and will be conducted at approximately 40 sites in the U.S. and Europe. The primary endpoints will be similar to the OPTIC trial and focus on mean change in BCVA and CST from baseline to one year, and incidence and severity of adverse events. Other data points will include protein expression of aflibercept starting at 10 weeks and an interim analysis at 26 weeks. The study will also evaluate the effectiveness and tolerability of the prophylactic steroid regimens.
As an investigator in OPTIC, I have observed the potential of ADVM-022 to significantly reduce the treatment burden for my patients with neovascular age-relatedmacular degeneration. In the latest data from the OPTIC trial, we have seen a manageable safety profile, robust aflibercept expression, and sustained anatomical improvements after two years following a single 2E11 dose of ADVM-022, said Dante Pieramici, M.D., partner, California Retina Consultants, and presenter of the data at ASRS. I look forward to participating as a LUNA investigator and building on the efficacy and safety profile in OPTIC with a new, lower 6E10 dose and to determine an optimal prophylactic steroid regimen for patients.
About Wet Age-Related Macular Degeneration
Wet AMD, also known as neovascular AMD or nAMD, is an advanced form of AMD, affecting approximately 10% of patients living with AMD. Wet AMD is a leading cause of blindness in patients over 65 years of age, with a prevalence of approximately 20 million individuals worldwide living with this condition. The incidence of new cases of wet AMD is expected to grow significantly worldwide as populations age. AMD is expected to impact 288 million people worldwide by 2040, with wet AMD accounting for approximately 10% of those cases.
About OPTIC Trial of ADVM-022 in Wet AMD
ADVM-022 is Adverums clinical-stage gene therapy product candidate being developed for the treatment of wet AMD. ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require a surgery to administer the gene therapy under the retina (sub-retinal approach), ADVM-022 has the advantage of being administered as a one-time IVT injection in the office and is designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance, and improve vision outcomes for patients with wet AMD.
The OPTIC trial is designed as a multi-center, open-label, dose-ranging, safety and efficacy trial of ADVM-022 in patients with wet AMD who have demonstrated responsiveness to anti-VEGF treatment. Patients in OPTIC are treatment-experienced, and previously required frequent anti-VEGF injections to manage their wet AMD and to maintain functional vision.
About Adverum Biotechnologies
Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for a number of highly prevalent ocular diseases with the aspiration of developing functional cures for these diseases to restore vision and prevent blindness. Leveraging the research capabilities of its proprietary, intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, ixoberogene soroparvovec (Ixo-vec), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. By overcoming the challenges associated with current treatment paradigms for these debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit http://www.adverum.com.
Forward-looking Statements
Statements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding Adverums plans to initiate a Phase 2 study in wet AMD to investigate the 2x10^11 vg/eye dose and a lower 6x10^10 vg/eye dose of ADVM-022, as well as new enhanced prophylactic steroid regimens, including local steroids and a combination of local and systemic steroids, planned for the third quarter of 2022, as well as the benefits Adverum expects from this trial, and the timing of preliminary data from the LUNA trial. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; enrollment uncertainties the results of early clinical trials not always being predictive of future clinical trials and results; and the potential for future complications or side effects in connection with use of ADVM-022. Additional risks and uncertainties facing Adverum are set forth under the caption Risk Factors and elsewhere in Adverums Securities and Exchange Commission (SEC) filings and reports, including Adverums Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 filed with the SEC on May 12, 2022. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Corporate & Investor Inquiries
Anand ReddiVice President, Head of Corporate Strategy and External Affairs & EngagementAdverum Biotechnologies, Inc.T: 650-649-1358E: areddi@adverum.com
Media
Megan TalonAssociate Director, Corporate CommunicationsAdverum Biotechnologies, Inc.T: 650-649-1006E: mtalon@adverum.com
Global Osteoarthritis Gene Therapy Market 2022 Industry Growth and Product Scope 2028 Top Players as Medipost, Mundipharma Travel Adventure Cinema -…
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The report presents decisive clarity towards highlighting the most effective segment that enables heavy revenue flow. Relevant details about other market segments are also discussed in the report to show logical conclusions about the most prominent segments in the global Osteoarthritis Gene Therapy market. The report is divided into various segments such as by-products, applications, and regions which will give a universal view of the market growth.
The well-established players in the global Osteoarthritis Gene Therapy market are:
In market segmentation by types, the report covers:
In market segmentation by applications, the report covers the following uses:
Major regions discussed in the research report of the market include:
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The pointers covered in the market dynamics are the drivers, restraints, and opportunities which affect the growth of the global Osteoarthritis Gene Therapy market during 2022 to 2028 period. The global regional analysis of the market was conducted and is mentioned in the report. Moreover, all the key pointers are explained briefly on a regional basis. The report also includes Porters Five Forces model to give the competitive scenario of the global Osteoarthritis Gene Therapy market.
The report covers expected revenue growth, potential lucrative opportunities, product ranges, pricing factors, and parameters to confer the emerging and new entrants in the industry with structured market data. The report caters to know the dynamics of the global Osteoarthritis Gene Therapy market opportunities and anticipate change.
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Global Osteoarthritis Gene Therapy Market 2022 Industry Growth and Product Scope 2028 Top Players as Medipost, Mundipharma Travel Adventure Cinema -...
Gene Synthesis Market Registered Substantial CAGR Growth Of 27.0% Forecast to 2029 and Market Is Analyzed By Size, Trends, Analysis, Future Scope,…
Gene Synthesis Market report has a lot of features to offer for Gene Synthesis industry which includes general market conditions, trends, inclinations, key players, opportunities, and geographical analysis. This report emphasizes on changing dynamics, growth-driving factors, restraints, and limitations. It includes a detailed analysis of the Gene Synthesis market in customer requirements, customer preferences, and the competitive landscape of the overall market. This report employs SWOT analysis technique for the assessment of the development of the most remarkable market players. An influential Gene Synthesis market report offers broader perspective of the market place with its comprehensive market insights and analysis.
Gene Synthesis business report makes available an overview of the Gene Synthesis industry which is gaining momentum in the last few years. The market report uses a range of steps for collecting, recording, analysing and interpreting market data to make this report all-inclusive. It also offers an outline of the industry that might promote interest among prospective investors, large corporations and everyday users who could participate in the next big opportunity or make their lives just a little easier. The world class Gene Synthesis report comprises of the list of leading competitors, strategic industry analysis and the insights of key factors influencing the Gene Synthesis industry.
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Data Bridge Market Research analyses that the market is growing with a CAGR of 27.0% in the forecast period and is expected to reach USD 21,156.93 million by 2028. Rising prevalence of infectious diseases demands the novel and advanced gene therapy products, vaccines, personalized medicines among others which can be achieved by using gene sequences, thus acts as driver for the gene synthesis market growth.
Gene synthesisis technique of synthetic biology which is employed for the production or creation of artificial gene under laboratory conditions. Gene synthesis technique is used for creating fusion proteins, molecular cloning or in order to achieve high level of protein expression. Traditionally, molecular cloning techniques used which consumes a lot of time and requires a lot of money investment.
Rise in adoption of gene therapy has enhanced the requirement of rapid gene synthesis products which provides with error free gene sequences in less interval of time and hence acts as driver for the gene synthesis market. The ethical issues associated with genetic engineering lowers the demand of gene constructs having the potential to interfere with organisms genomic composition which is restraining the gene synthesis market growth. Increasing demand of personalized medicines has paved the way for genomic which demands the error free gene sequences in at low cost in less span of time, thus acts as an opportunity for the gene synthesis market. Long approval time for gene synthesizer and products synthesized through these products restrict the product manufacturers to earn potential benefit in the market and impacts the overall market size, hence acts as challenge for the gene synthesis market growth.
Market Players Covered:
Eurofins Genomics, ATG:biosynthetics GmbH, Merck KGaA, Aldevron, Synbio Technologies, Thermo Fisher Scientific Inc, Genscript, General Biosystems, Twist Bioscience, Evonetix, Bio-synthesis Inc, LGC Limited, Eurofins Genomics Blue Heron LLC, Macrogen,Inc, Science Exchange, Inc, Integrated DNA Technologies, Inc, Origene Technologies, INC., Proteo Genix, Biocat GMBH among other domestic players. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.
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The gene synthesis market is analysed and market size information is provided by the country, product & services, gene type, product type, application, method, end user and distribution channel as referenced above.
The countries covered in the gene synthesis market report are the U.S., Canada and Mexico, U.K., Germany, France, Spain, Italy, Netherlands, Switzerland, Russia, Turkey, Austria, Ireland, and Rest of Europe, China, South Korea, Japan, India, Australia, Singapore, Malaysia, Indonesia, Thailand, Philippines and rest of Asia-Pacific, South Africa, Saudi Arabia, U.A.E., Israel, Egypt and rest of Middle East and Africa, Brazil, Argentina, and rest of South America.
Software and services segment.in North American region is expected to grow with the highest growth rate in the forecast period of 2021 to 2028 because of increasing gene therapy based medications. The software and services segment in Germany is dominating the European market owing to increasing research and development for the gene synthesis used in vaccine preparation. China is leading the growth of the Asia-Pacific market and Software and services segment is dominating in this country due to presence of highest number of gene synthesis providers.
The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.
Highlights of TOC:
Chapter 1: Market overview
Chapter 2: Global Gene Synthesis Market
Chapter 3: Regional analysis of the Global Gene Synthesis Market industry
Chapter 4: Gene Synthesis Market segmentation based on types and applications
Chapter 5: Revenue analysis based on types and applications
Chapter 6: Market share
Chapter 7: Competitive Landscape
Chapter 8: Drivers, Restraints, Challenges, and Opportunities
Chapter 9: Gross Margin and Price Analysis
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Highlights of Following Key Factors of Global Gene Synthesis Market
A detailed description of the companys operations and business divisions
Analysts summarization of the companys business strategy
Progression of key events associated with the company
A list of major products, services and brands of the company
A list of key competitors to the company
A list and contact details of key locations and subsidiaries of the company
The latest financial ratios derived from the annual financial statements published by the company with 5 years history.
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Will 100 Million Die From the COVID Vax by 2028? – The Epoch Times
Via this genetic engineering experiment, theyve literally injected seeds of demise into everyday people like a cockroach spray. Based on a 2011 estimate, he believes an extra 700 million will be killed from this bioweapon and theyve known about the risks since 2005.
In this revealing interview with Greg Hunter of USAWatchdog.com, David Martin, Ph.D., presents evidence that COVID-19 injections are not vaccines but bioweapons that are being used as a form of genocide across the global population.1
In March 2022, Martin filed a federal lawsuit against President Biden, the Department of Health and Human Services and the Centers for Medicare and Medicaid Services alleging that COVID-19 shots turn the body into a biological weapons factory, manufacturing spike protein. Not only is the term vaccination misleading when referring to COVID-19 shots, its inaccurate since they are actually a form of gene therapy.2
And we are not only not going to be sued for, you know, any libel or misinformation, we are actually holding people criminally accountable for their domestic terrorism, their crimes against humanity and the story of the coronavirus weaponization that goes back to 1998, Martin says.3
Martin has been in the business of tracking patent applications and approvals since 1998. His company, M-Cam International Innovation Risk Management, is the worlds largest underwriter of intangible assets used in finance in 168 countries. M-Cam has also monitored biological and chemical weapons treaty violations on behalf of the U.S. government, following the anthrax scare in September 2001.4
According to Martin, there are more than 4,000 patents relating to the SARS coronavirus. His company has also done a comprehensive review of the financing of research involving the manipulation of coronaviruses that gave rise to SARS as a subclade of the beta coronavirus family.
Much of the research was funded by the National Institutes of Allergy and Infectious Diseases (NIAID) under the direction of Dr. Anthony Fauci.5Martin explained:6
I think its important for your listeners and viewers to remember that it was 1999 when Anthony Fauci and Ralph Baric at the University of North Carolina Chapel Hill decided to start weaponizing coronavirus they patented in 2002 and you heard that date correctly, thats a year before the SARS outbreak in China.
You know, they knew it was a bioweapon since 2005. They knew it was effective at harming populations, intimidating and coercing populations
According to Martin, the spike protein that the COVID-19 shots manufacture is a computer simulation of a chimera of the spike protein of coronavirus. It is, in fact, not a coronavirus vaccine. It is a spike protein instruction to make the human body produce a toxin, and that toxin has been scheduled as a known biologic agent of concern with respect to biological weapons for the last now decade and a half, he said.7
Rather than being a public health measure as they were widely campaigned to be, COVID-19 shots are an act of bioweapons and bioterrorism. Martin shared that in 2015, Dr. Peter Daszak, head of the EcoHealth Alliance that funneled research dollars from the NIAID to the Wuhan Institute of Virology for coronavirus research, stated:8
We need to increase public understanding of the need for medical countermeasures such as a pan-coronavirus vaccine. A key driver is the media and the economics will follow the hype. We need to use that hype to our advantage, to get to the real issues. Investors will respond if they see profit at the end of the process.
Daszak, who Martin refers to as the money launderer in chief, actually stated that this entire exercise was a campaign of domestic terror to get the public to accept the universal vaccine platform using a known biological weapon. And that is their own words, not my interpretation, Martin said.9
Both Pfizer and Modernas COVID-19 shots contain nucleic acid sequences that are not part of nature and have not been previously introduced to the human body. This amounts to a genetic engineering experiment that did not go through animal studies or clinical trials.
However, already people are dying from the shots and, Martin states, many more will due to issues such as blood clots, damage to the cardiovascular system and problems with liver, kidney and pulmonary function.10
An onslaught of reproductive and cancer cases related to the shots are also anticipated. The fact of the matter is an enormous number of people who are injected are already carrying the seeds of their own demise, Martin said.11As for how many may die, Martin believes the numbers may have been revealed back in 2011, when the World Health Organization announced their decade of vaccination:12
Based on their own 2011 estimate, and this is a chilling estimate, but we just have to put it out there When the Bill and Melinda Gates Foundation, the Chinese CDC, the Jeremy Farrar Wellcome Trust and others published The Decade of Vaccination for the World Health Organization back in 2011 their stated objective was a population reduction of 15% of the worlds population.
Put that in perspective, thats about 700 million people dead and that would put the U.S. participation in that certainly as a pro rata of injected population somewhere between 75 and 100 million people.
When asked what timeframe these people may die in, Martin suggested theres a lot of economic reasons why people hope that its between now and 2028.13This is because of a tiny little glitch on the horizon the projected illiquidity of the Social Security, Medicare and Medicaid programs by 2028.
So the fewer people who are recipients of Social Security, Medicare and Medicaid, the better, Martin said. Not surprisingly, its probably one of the motivations that led to the recommendation that people over the age of 65 were the first ones getting injected.14Other populations at risk are caregivers, including health care providers, and others in the workforce who were forced to be injected, such as pilots.
Why is it that were suddenly having 700 flights a day being canceled because, allegedly, airlines dont have pilots? the dirty secret is there a lot of pilots who are having microvascular problems and clotting problems, and that keeps them out of the cockpit, which is a good place to not have them if theyre going to throw a clot for a stroke or a heart attack,Martin said.
But the problem is were going to start seeing that exact same phenomenon in the health care industry and at a much larger scale, which means we now have, in addition to the problem of the actual morbidity and mortality, meaning people getting sick and people dying.
We actually have that targeting the health care industry writ large, which means we are going to have doctors and nurses who are going to be among the sick and the dead. And that means that the sick and the dying also do not get care.15
Its been stressed by the media and public health officials that COVID-19 shots do not alter DNA. However, Martin brings attention to a little-known grant from the National Science Foundation, known as Darwinian chemical systems,16which involved research to incorporate mRNA into targeted genomes. According to Martin:17
Moderna was started on the back of a 10-year National Science Foundation grant. And that grant was called Darwinian chemical systems the project that gave rise to the Moderna company itself was a project where they were specifically figuring out how to get mRNA to write itself into the genome of whatever target they were going after.
That could be a single-celled organism, it could be a multi-celled organism or it could be a human. And the fact of the matter is Moderna was started on the back of having proven that mRNA can be transfected and write itself into the human genome.
It is completely unknown what the short- or long-term effects of the spike protein analog thats inside people who received COVID-19 injections will be. But with respect to alteration of the genome, Martin states that data show mRNA has the capacity to write into the DNA of humans, and as such, the long-term effects are not going to merely be symptomatic. The long-term effects are going to be the human genome of injected individuals is going to be altered.18
The 2001 anthrax attack, which came out of medical and defense research, led to the passage of the PREP Act, which removed liability for manufacturers of emergency medical countermeasures.
This means that as long as the U.S. is under a state of emergency, things like COVID-19 vaccines are allowed under emergency use authorization. And as long as the emergency use authorization is in effect, the makers of these experimental gene therapies are not financially liable for any harm that comes from their use.
That is, provided theyre vaccines. If these injections are NOT vaccines, then the liability shield falls away, because there is no liability shield for a medical emergency countermeasure that is gene therapy. Further, lawsuits that can prove the companies engaged in fraud will also negate the liability shield. Martin states:19
One of the convenient things about the PREP Act is the immunity shield from liability actually is only as good as the absence of fraud. Because if there was fraud in the promulgation of the events, leading to an emergency use authorization, then all of the immunity shield gets wiped out.
So the reason why it is so important for conversations like the one were having to actually be promoted and be advanced is because the pharmaceutical companies and this includes Pfizer and Moderna and J&J know they are perpetuating a fraud. The great thing about this is when that fraud is established, 100% of the liability flows back to them.
when a fraud was the basis for a fraud, then we actually have a number of other legal remedies that allow you to pierce that veil. So in the end, theres no question and its quite evident based on the current mortality and morbidity data that given the fact that when it comes to biological weapons and bioterror each count comes with $100 million penalty. Thats what the federal statute gives us.
The penalty for corporate domestic terrorism, when you have per count $100 million a pop liabilities that is an existential threat that takes a company like Pfizer or takes a company like Moderna out of existence. And that is what were working for every day.
If youd like to follow the progress of the ongoing legal cases seeking to expose the truth that a criminal organization is seeking to obtain control over the global population via the creation of patented bioweapons marketed as novel viruses and injections you can find all the details at ProsecuteNow.io, a website compiled by Martin and colleagues.20
Originally publishedJuly 16, 2022 on Mercola.com
Views expressed in this article are the opinions of the author and do not necessarily reflect the views of The Epoch Times. Epoch Health welcomes professional discussion and friendly debate. To submit an opinion piece, please follow these guidelines and submit through our form here.
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Will 100 Million Die From the COVID Vax by 2028? - The Epoch Times
Comparing 4D Molecular Therapeutics (FDMT) and Its Rivals – Defense World
4D Molecular Therapeutics (NASDAQ:FDMT Get Rating) is one of 264 public companies in the Biological products, except diagnostic industry, but how does it weigh in compared to its peers? We will compare 4D Molecular Therapeutics to similar companies based on the strength of its institutional ownership, analyst recommendations, earnings, valuation, profitability, risk and dividends.
This table compares 4D Molecular Therapeutics and its peers net margins, return on equity and return on assets.
76.6% of 4D Molecular Therapeutics shares are owned by institutional investors. Comparatively, 53.5% of shares of all Biological products, except diagnostic companies are owned by institutional investors. 11.2% of 4D Molecular Therapeutics shares are owned by company insiders. Comparatively, 16.4% of shares of all Biological products, except diagnostic companies are owned by company insiders. Strong institutional ownership is an indication that large money managers, endowments and hedge funds believe a stock will outperform the market over the long term.
4D Molecular Therapeutics peers have higher revenue and earnings than 4D Molecular Therapeutics. 4D Molecular Therapeutics is trading at a lower price-to-earnings ratio than its peers, indicating that it is currently more affordable than other companies in its industry.
This is a summary of current ratings and price targets for 4D Molecular Therapeutics and its peers, as provided by MarketBeat.
4D Molecular Therapeutics presently has a consensus target price of $26.00, suggesting a potential upside of 157.94%. As a group, Biological products, except diagnostic companies have a potential upside of 92.79%. Given 4D Molecular Therapeutics stronger consensus rating and higher possible upside, analysts clearly believe 4D Molecular Therapeutics is more favorable than its peers.
4D Molecular Therapeutics has a beta of 1.26, meaning that its stock price is 26% more volatile than the S&P 500. Comparatively, 4D Molecular Therapeutics peers have a beta of 0.75, meaning that their average stock price is 25% less volatile than the S&P 500.
4D Molecular Therapeutics beats its peers on 8 of the 13 factors compared.
(Get Rating)
4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company, develops product candidates using its adeno-associated viruses vectors. It develops a portfolio of gene therapy product candidates focuses in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The company has three product candidates that are in clinical trials: 4D-125 that is in a Phase 1/2 clinical trial for the treatment of X-linked retinitis pigmentosa; 4D-110 that is in a Phase 1/2 clinical trial for the treatment of choroideremia; and 4D-310, which is in a Phase 1/2 clinical trial for the treatment of Fabry disease. Its two IND candidates are 4D-150 for the treatment of wet age-related macular degeneration and 4D-710 for the treatment of cystic fibrosis lung disease. 4D Molecular Therapeutics, Inc. has research and collaboration arrangements with uniQure; CRF; Roche; and CFF. The company was founded in 2013 and is headquartered in Emeryville, California.4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company, develops product candidates using its adeno-associated viruses vectors. It develops a portfolio of gene therapy product candidates focuses in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The company has three product candidates that are in clinical trials: 4D-125 that is in a Phase 1/2 clinical trial for the treatment of X-linked retinitis pigmentosa; 4D-110 that is in a Phase 1/2 clinical trial for the treatment of choroideremia; and 4D-310, which is in a Phase 1/2 clinical trial for the treatment of Fabry disease. Its two IND candidates are 4D-150 for the treatment of wet age-related macular degeneration and 4D-710 for the treatment of cystic fibrosis lung disease. 4D Molecular Therapeutics, Inc. has research and collaboration arrangements with uniQure; CRF; Roche; and CFF. The company was founded in 2013 and is headquartered in Emeryville, California.
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Comparing 4D Molecular Therapeutics (FDMT) and Its Rivals - Defense World
Boyle Heights Selected as Host Site for Artificial Intelligence Bootcamp APPLICATIONS NOW OPEN! – GlobeNewswire
BOYLE HEIGHTS, Calif., July 14, 2022 (GLOBE NEWSWIRE) -- Global biotherapeutics leader CSL Behring will host a Mark Cuban Foundation Artificial Intelligence (AI) Bootcamp for high school students in the Fall of 2022 in conjunction with the i.am/Angel Foundation. The No Cost AI Bootcamp in Boyle Heights will be targeted at high school students and will introduce underserved students in grades 9 through 12 to basic AI concepts and skills.
CSL Behring is one of 20+ host companies selected to host 30 camps across the US in Fall 2022.
The CSL Behring Bootcamp will be held over four consecutive Saturdays starting on October 22nd and ending on November 12th.
The student and parent application are now open at markcubanai.org/application. Students do not need any prior experience with computer science, programming, or robotics to apply and attend.
Throughout the AI Bootcamp, students will learn what artificial intelligence is and is not, where they already interact with AI in their own lives, and the ethical implications of AI systems including but not limited to TikTok recommendations, smart home assistants, facial recognition, and self-driving cars to name a few.
Students will benefit from volunteer mentor instructors who are knowledgeable about data science and able to help students quickly understand material normally taught at a collegiate level. As part of the 5-hour curriculum, students also get to work in Microsoft Azure, Microsoft's cloud computing platform, each day to build their own AI applications related to Chatbots, Computer Vision, Machine Learning, and Natural Language Processing.
The Mark Cuban Foundation provides the bootcamps curriculum materials, trains volunteer mentors, and recruits and selects local students to attend camp. In addition, the Mark Cuban Foundation and CSL Behring work together to provide food, transportation, and access to laptops for students at no cost throughout the duration of Bootcamp.
"This bootcamp was a great experience and opened my eyes to the multitude of ways that AI can be used. Would definitely recommend it!" - Nhaomi L., CSL Behring 2021
Founded by Mark Cuban in 2019, the AI Bootcamp initiative has hosted free AI bootcamps for students across several US cities, including Dallas, Pasadena, Chicago, Pittsburgh, Detroit, and Atlantic City to name a few. The Mark Cuban Foundation has impacted 450+ students to date and has a goal to have 1,000 students graduate from AI Bootcamps Program in 2023.
CSL Behring develops and delivers innovative therapies for people living with rare and serious conditions. The companys global cell & gene therapy research hub is located in nearby Pasadena.
Students interested in applying to the Mark Cuban Foundation AI Bootcamp should do so before Thursday, September 1st, 2022 at markcubanai.org/application. To see our 2022 camp locations and to learn more about the Mark Cuban Foundation AI Bootcamps, please visit markcubanai.org/faq.
Contact: Carli Lidiak, Mark Cuban FoundationPhone: 309-840-0348Email: carli.lidiak@markcubanai.org
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Boyle Heights Selected as Host Site for Artificial Intelligence Bootcamp APPLICATIONS NOW OPEN! - GlobeNewswire
CRISPR in the Classroom – The New York Times
A decade after CRISPR started to become a major tool in genetic research, a new generation of scientists is growing up with the technology. Even high school students are able to run CRISPR experiments. Some specialized public high schools teach CRISPR as a hands-on lesson in biotechnology. These classes cover everything from molecular biology to pipetting to biomedical ethics and career options.
Visualizing and comprehending whats happening on the molecular level is usually always the challenge, said Katy Gazda, a high school biotechnology teacher who taught CRISPR in her classroom last year. To help students better understand complex molecular movements, teachers use tools like paper models, 3-D printed models and online animations.
Note: Diagram is simplified.
1. Target the right gene
Scientists engineer a piece of RNA that is a match for the DNA they want to edit. This is called the guide RNA. Students can practice designing their own guide RNA sequences using the same free bioinformatics search tool that scientists use for research.
2. Bind the target
An enzyme called Cas9 binds to a piece of DNA and temporarily unwinds a section of the DNA. Students can model the process with paper cutouts, pushing a paper DNA sequence along a printed guide RNA until they match.
3. Cut the DNA
If the guide RNA matches a section of the DNA, the Cas9 enzyme cuts both strands of the DNA double helix. An interactive animation from the Howard Hughes Medical Institute shows students how the Cas9 enzyme changes its shape and snips through the DNA.
Repaired DNA with edited section
DNA fragments cut by Cas9
DNA fragments cut by Cas9
Repaired DNA with edited section
Note: Diagrams are simplified.
4. Repair and edit the DNA
Machinery inside the cell rushes to fix the broken DNA. One repair process uses a similar-looking, unbroken piece of DNA as a template to stitch the broken pieces back together.
Scientists can introduce tailor-made DNA into the cell tricking the repair machinery into using the engineered DNA as the template for stitching together the broken pieces.
Students also learn about real-life examples of DNA editing, such as therapies for genetic illnesses including sickle cell disease and cystic fibrosis. In some exercises, they can see examples of the actual DNA sequence associated with each illness and evaluate specific gene edits proposed to cure the illness.
Ms. Gazda believes that hands-on lab lessons help students open their minds to the idea that they can truly be a scientist. Several companies sell CRISPR curriculum kits to high schools and universities. One kit from Bio-Rad, a life science technology manufacturer, includes a prepackaged experiment using E. coli bacteria.
Bacteria Altered With CRISPR
E. coli
bacteria colonies
Petri
dishes full of
bacteria food
Petri
dish full of
bacteria food
Bacteria Altered With CRISPR
Petri
dish full of
bacteria food
A gene in the bacteria encodes an enzyme, called -gal, which can help break down certain molecules.
A bright blue color appears when X-gal is broken down by -gal.
A gene in the bacteria encodes an enzyme, called -gal, which can help break down certain molecules.
A bright blue color
appears when X-gal is
broken down by -gal.
Note: Diagrams and molecule names are simplified.
The E. coli bacteria used in the Bio-Rad kit are grown on a food mixture that includes a compound called X-gal. Normally, these bacteria are able to use an enzyme to break down the compound into two parts: a sugar molecule and an indicator molecule.
The indicator molecule turns deep blue, showing students that the bacterial enzyme is working. This colorful display is an important part of the experiment. It shows students what to expect in an unaltered, or control group a vital part of any scientific experiment.
The -gal enzyme is not produced.
The X-gal compound cannot be broken down without the -gal enzyme, so the bright blue indicator molecule is never produced.
After the students use CRISPR to transform a section of the gene, the gene is no longer functional.
After the students use CRISPR to transform a section of the gene, the gene is no longer functional.
The -gal enzyme is not produced. The X-gal compound cannot be broken down without the -gal enzyme, so the bright blue indicator molecule is never produced.
Note: Diagrams and molecule names are simplified.
The process of altering E. coli DNA with CRISPR involves lab techniques like pipetting liquids and carefully moving bacteria colonies. Teaching a new lab class like CRISPR can be intimidating, says Gregory Jubulis, a high school science teacher who uses the Bio-Rad kit in his biotechnology class. It takes you a few years before youre real comfortable with teaching something, he said.
But when classroom CRISPR lab kits first became available, he knew he wanted to teach it. I just want my kids to be ready for the future of science, he said.
Ms. Gazda uses lab classes as an opportunity to share career options with students. In a CRISPR lab, students may learn about careers in anything from molecular and cellular biology to entrepreneurship and science journalism.
Ethics always comes up, says Mr. Jubulis, explaining how he relates the lab experiment to real-life CRISPR applications like gene therapy. Many of his students have friends or family with genetic illnesses, so the topic can be deeply personal.
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CRISPR in the Classroom - The New York Times
UNE researcher awarded $1.8 million to study chronic pain relief through gene therapy – University of New England
Chronic pain affects millions of people worldwide, yet popular treatments for pain including surgery and opioid medications can have disastrous side effects of their own. But with $1.8 million in funding from the National Institute of Neurological Disorders and Stroke (NINDS), a University of New England researcher will explore non-opioid treatments for chronic pain at the cellular level.
Benjamin Harrison, B.Sc., Ph.D., assistant professor of biochemistry and nutrition, will use the five-year R01 grant from the National Institutes of Health to study how to reduce the excitability of nociceptors, which are neurons that transmit pain signals in response to painful injuries.
Harrison and his team have discovered that nociceptors contain a protein called "CELF4, an RNA binding protein they theorize inhibits the production of pro-nociceptive, or pro-pain-sensing, cellular components. Harrisons research will focus on delivering CELF4 into pain neurons, where this protein will limit the synthesis of ion-channels, receptors, and other molecules that sensitize them.
Specifically, the researchers will study if a locally administered adeno-associated virus can stimulate production of CELF4 and reduce pain in those areas an approach known as gene vector therapy.
Harrison remarked that the innovative approach could prove beneficial for those living with chronic pain but who do not want to undergo surgeries which can be expensive and leave people with no sensation at all or use powerful pain-reducing medications like addictive opioids.
There are some chronic pain conditions that are simply intolerable, and people with those conditions are willing to do severe surgeries to reduce their pain, Harrison remarked. Using this novel gene therapy vector approach, we can develop pain therapies that are less invasive than surgery and carry fewer risks than conventional opioid medications.
Future directions for the research could include partnerships with clinicians for clinical trials, Harrison said.
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UNE researcher awarded $1.8 million to study chronic pain relief through gene therapy - University of New England
SwanBio Presents Design of Innovative Natural History Study Aimed to Evolve Understanding of Adrenomyeloneuropathy and Inform Future Treatments -…
PHILADELPHIA--(BUSINESS WIRE)-- SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today presented details about the companys ongoing natural history study of adrenomyeloneuropathy (AMN) at the 8th Congress of the European Academy of Neurology (EAN) in Vienna. The intention of the CYGNET study is to assess disease progression in patients with AMN to inform the research and development of potential treatments, including SwanBios lead candidate, SBT101, the first clinical-stage AAV-based gene therapy for AMN.
At SwanBio, our approach to drug development begins with deepening our understanding of the origin and trajectory of diseases like AMN, ideally enabling us to deliver gene therapies that are both highly effective and meaningful for the quality of life of patients. With this in mind, our proprietary CYGNET study was designed to better characterize AMN disease progression, which varies from person to person, said Steven Zelenkofske, D.O., chief medical officer, SwanBio Therapeutics. CYGNET is the first AMN clinical study to feature wearables, which may help us identify sensitive outcomes related to clinically relevant changes early in men with AMN.
SwanBios CYGNET natural history study will track a number of variables over a two-year period, including:
More details about the CYGNET study, including demographics and disease characteristics of the first 21 patients enrolled, were presented during a virtual poster session at EAN.
SwanBio anticipates recruiting approximately 80 patients for the CYGNET natural history study; as of early June, the study was over 40% enrolled across five different global sites. Clinicians or patients interested in learning more about this study can review SwanBios CYGNET flyer or contact SwanBio at clinicaltrials@swanbiotx.com.
Presentation Details ePoster EPO-599: CYGNET: A prospective multicenter observational study of disease progression in patients with adrenomyeloneuropathyDate & Time: June 27, 2022, 12:30 1:15 p.m. CEST
About SBT101 SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people with adrenomyeloneuropathy (AMN). SBT101 was designed to compensate for the disease-causing ABCD1 mutation. In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of disease markers and functional improvement in AMN mouse models. SBT101 was also shown to be well-tolerated in non-human primates at six months post-treatment. The clinical program for SBT101 builds on this positive preclinical data, plus the companys already deep understanding of the underlying pathophysiology of the disease and the patient experience of AMN.
SwanBio expects to initiate a randomized, controlled Phase 1/2 clinical trial designed to assess the safety and efficacy of SBT101 in patients with AMN in the second half of 2022. In early 2022, the FDA cleared SwanBios Investigational New Drug application for SBT101 and granted SBT101 Fast Track and Orphan Drug Designation.
About Adrenomyeloneuropathy Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction, which all affect quality of life. Between 8,000-10,000 men in the United States and European Union are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom management.
About SwanBio Therapeutics SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com.
References
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Gene and Cell Therapy (GCT) Market Report (COVID-19 Analysis) by Worldwide Market Trends & Opportunities and Forecast to 2030 Designer Women -…
The Global Gene and Cell Therapy (GCT) Market is estimated at USD 2504.2 Million in the year 2020. Growth in the historic period in the cell and gene therapy market resulted from increase in investments in cell and gene therapies, growth in research and development, advances in cancer drug discovery, rise in public-private partnerships, strong economic growth in emerging markets, increased healthcare expenditure, and rising in pharmaceutical R&D expenditure.
Companies in the gene and cell therapy for oncology market are increasing their product innovation through strategic collaborations. To sustain in the increasingly competitive market, organizations are developing innovative products as well as sharing skills and expertise with other such enterprises. While oncology drug companies have long collaborated with each other as well as with academic and research institutions in this market by way of partnerships, in or out licensing deals, this trend has been increasing over the recent years.
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Further, the market was restrained by inadequate reimbursements, challenges due to regulatory changes, low healthcare access, and limited number of treatment centers. Going forward, increasing prevalence of cancer and chronic diseases, rising geriatric population, rising focus on cell and gene therapy, and rise in healthcare expenditure will drive the growth in the gene and cell therapy market. Factors that could hinder the growth of the market in the future include high costs of therapy, stringent regulations, reimbursement challenges, and coronavirus pandemic.
Scope of the Report
The report presents the analysis of Gene and Cell Therapy market for the historical period 2016-2020 and forecast period of 2021-2026.The report analyses the Gene and Cell Therapy Market by value (USD Million).The report analyses the Gene and Cell Therapy Market by Vector (Lentivirus, AAV, Retrovirus & Gammaretrovirus and Others).The report analyses the Gene and Cell Therapy Market Application (Oncology, Neurological disorders, cardiovascular disorders, Others).The Global Gene and Cell Therapy Market has been analysed By Region (North America, Europe, Asia Pacific, LAMEA), By Country (United States, Canada, Germany, U.K, France, Italy, China, Japan, India and South Korea).The key insights of the report have been presented through the frameworks of the attractiveness of the market has been presented by region, by Vector, By Application.
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Also, the major opportunities, trends, drivers and challenges of the industry has been analysed in the report.The report tracks competitive developments, strategies, mergers and acquisitions and new product development. The companies analysed in the report include F. Hoffman-La Roche Ltd., Novartis, Sanofi, Alnylam Pharmaceuticals Inc., Pfizer, BlueBird Inc., Sarepta Therapeutics, Voyager Therapeutics, Orchard Therapeutics Plc, AnGes Inc.
Key Target AudienceBiotechnology CompaniesPharmaceutical and Health CompaniesConsulting and Advisory FirmsGovernment and Policy MakersRegulatory Authorities
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Surgeon taking part in gene therapy trial to slow vision loss – KCRA Sacramento
that's the center of the macula right there. Okay, so if you had an area of degeneration over here or over here or over here, you would have no symptoms. But it's that center part that dr eric swan with the retina care center says causes vision loss once affected. And it can be *** little or *** lot depending on how much tissue has been lost due to age related dry macular degeneration. Typical patient comes to see me has the loss of central vision, they just can't read. They're having difficulties driving. And so this is *** huge impairment for patients because the tissue can't be regenerated. The goal is to slow progression. Dr swan is part of *** nationwide FDA approved investigative clinical trial. That involves *** one time surgery using *** patient's own cells to create more of *** certain protein. Thought to be low in patients with this eye disease. This is *** very novel technique. It's gene therapy. So we're actually injecting these genes underneath the retina and telling the cells to produce more of this protein. Dr swan is the first surgeon in Maryland to perform the surgery and says clinical trials are still in the early stages. Early detection is key and that's why exams he says are so important and why research is so crucial when it comes to quality of life issues like vision. It is I mean, you want your grandmother your love when your grandfather to actually be able to continue to drive and really to maintain their independence and that really is the most important for them, jennifer francie adi W. B. *** l tv 11 News.
Surgeon taking part in gene therapy trial to slow vision loss
Updated: 7:26 PM PDT Jun 25, 2022
A Baltimore doctor performed the first surgery in Maryland as part of a nationwide clinical trial to slow the progression of what's known as dry macular degeneration.Dr. Eric Suan, retinal surgeon at the Retina Care Center, said degeneration causes vision loss once affected. And it can be a little or a lot, depending on how much tissue has been lost due to age-related dry macular degeneration."The typical patient who comes to me has a loss of central vision," Suan said. "They just can't read, they're having difficulty driving, and so this is a huge impairment for patients."Because the tissue can't be regenerated, the goal is to slow progression.Suan is part of a nationwide Food and Drug Administration-approved investigative clinical trial that involves a one-time surgery using a patient's own cells to create more of a certain protein thought to be low in patients with this eye disease."This is a very novel technique. It's gene therapy. So, we are actually injecting these genes under the retina and telling the cells to produce more of this protein," Suan said.Suan said clinical trials are still in the early stages but that early detection is key, which makes eye exams important. Suan said research is crucial when it comes to a quality-of-life issue like vision."Do you want your grandmother and your loved one, grandfather, to actually be able to continue to drive to maintain independence? That really is the most important for them," Suan said.
A Baltimore doctor performed the first surgery in Maryland as part of a nationwide clinical trial to slow the progression of what's known as dry macular degeneration.
Dr. Eric Suan, retinal surgeon at the Retina Care Center, said degeneration causes vision loss once affected. And it can be a little or a lot, depending on how much tissue has been lost due to age-related dry macular degeneration.
"The typical patient who comes to me has a loss of central vision," Suan said. "They just can't read, they're having difficulty driving, and so this is a huge impairment for patients."
Because the tissue can't be regenerated, the goal is to slow progression.
Suan is part of a nationwide Food and Drug Administration-approved investigative clinical trial that involves a one-time surgery using a patient's own cells to create more of a certain protein thought to be low in patients with this eye disease.
"This is a very novel technique. It's gene therapy. So, we are actually injecting these genes under the retina and telling the cells to produce more of this protein," Suan said.
Suan said clinical trials are still in the early stages but that early detection is key, which makes eye exams important. Suan said research is crucial when it comes to a quality-of-life issue like vision.
"Do you want your grandmother and your loved one, grandfather, to actually be able to continue to drive to maintain independence? That really is the most important for them," Suan said.
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Surgeon taking part in gene therapy trial to slow vision loss - KCRA Sacramento
Ambys Medicines Announces Formation of Clinical and Scientific Advisory Boards with Leading Liver Disease and Cell and Gene Therapy Experts – Business…
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Ambys Medicines, a company pioneering cell-replacement therapies for patients with liver disease, today announced the formation of its clinical and scientific advisory boards comprising leading clinical experts in liver disease and hepatocyte transplantation, and world-class scientists pioneering cell and gene technologies.
The clinical advisory board provides guidance on advancing Ambyss lead program, AMI-918, through the clinic and provides critical input on clinical trial design and patient selection. The scientific advisory board will provide a cross-discipline perspective on applying cutting-edge technology to Ambyss cell therapy platform to progress its discovery pipeline of next generation cell therapies.
Were honored to work with such an esteemed and diverse group of experts in liver disease, hepatocyte transplantation, and cell and gene therapies whose collective experience will be highly valuable as we finalize our clinical development strategy for AMI-918 and progress our genetically engineered hepatocyte follow-on programs, said Ronald Park, M.D., Chief Executive Officer of Ambys Medicines. Were grateful for the engagement and support from our advisors as we work to bring first-in-class hepatocyte replacement therapies to liver failure patients who currently lack treatment options.
Each of our advisors brings incredible knowledge and expertise in their respective fields that will be instrumental to Ambys as we continue to broaden our pipeline and move closer to becoming a clinical-stage company, said Markus Grompe, M.D., Founder and Chief Scientific Officer of Ambys Medicines. Were excited to partner together to realize the potential of our novel replacement cell therapy platform in restoring lost hepatic function to patients with acute or chronic liver failure and genetic liver diseases.
Clinical Advisory Board
Scientific Advisory Board
About Ambys MedicinesAmbys Medicines is focused on pioneering cell replacement therapies for patients with liver failure. Ambyss proprietary platform enables the company to be the first and only company able to develop and manufacture functional human hepatocytes at scale. Our scientific approach has the potential to fundamentally transform the treatment paradigm for patients with acute and chronic liver failure and genetic diseases of the liver. Our lead program, AMI-918, is a hepatocyte replacement cell therapy in development to restore lost hepatic function. Beyond AMI-918, we are building a pipeline of next-generation modified hepatocytes that will rapidly expand the range of treatable patient populations. Learn more at ambys.com and follow us on Twitter, LinkedIn and Instagram.
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Ambys Medicines Announces Formation of Clinical and Scientific Advisory Boards with Leading Liver Disease and Cell and Gene Therapy Experts - Business...
Animal Stem Cell Therapy Market projected CAGR of 5.2% for the next ten years (2022-2032) Persistence Market Research – GlobeNewswire
New York, June 27, 2022 (GLOBE NEWSWIRE) -- The global animal stem cell therapy market recorded sales of around US$ 249.9 Mn in 2021 and the market is predicted to experience healthy growth over the years ahead at a CAGR of 5.2% (2022 to 2032).
Animal stem cell therapies are treatments for disorders such as arthritis, soft tissue injuries, traumatic fractures, tendonitis, inflammatory bowel disease, and others that are given to animals. Hematopoietic stem cells and mesenchymal stem cells are used in these treatment procedures.
As the focus on giving animals a better quality of life grows, so does the adoption of such therapies across the world. The market for animal stem cell therapy is predicted to continuously increase owing to the growing need for higher levels of veterinary care.
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Regulatory bodies are also encouraging veterinary regenerative medicine research & development.
The European Commission has approved a few stem cell-based products within the region.
The animal stem cell therapy market is moderately fragmented with a few key market players and presents numerous opportunities for new market entrants to create a foothold in the industry. Increased pet adoption rate can be a beneficial factor for manufacturers of stem cells to expand into emerging markets.
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With rising disposable incomes and higher adoption rate of pet animals, the global market for animal stem cell therapy is set to gain traction over the coming years, says an analyst of Persistence Market Research.
Market Competition
Key animal stem cell therapy providers are investing in innovating veterinary regenerative medicines and they are also coming up with new product launches for the well-being of animals.
Along with product innovation, market players are also aiming for various collaborations to strengthen their R&D in the field of animal stem cell therapy.
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What Does the Report Cover?
Persistence Market Research offers a unique perspective and actionable insights on the animal stem cell therapy market in its latest study, presenting historical demand assessment of 2012 2021 and projections for 2022 2032.
The research study is based on product type (hemopoietic stem cells and mesenchymal stem cells), source (allogeneic and autologous), indication (osteoarthritis, soft tissue injuries, traumatic fractures, tendonitis, inflammatory bowel disease, and others), species (canine, feline, and equine), and end user (veterinary hospitals, veterinary clinics, and veterinary research institutes), across seven key regions of the world.
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Animal Stem Cell Therapy Market projected CAGR of 5.2% for the next ten years (2022-2032) Persistence Market Research - GlobeNewswire
An Experimental Gene Therapy Changed His DNAAnd His Hair Color – The Atlantic
In October 2019, Jordan Janz became the first person in the world to receive an experimental therapy for cystinosis, a rare genetic disease. The treatment was physically grueling. Doctors extracted blood stem cells from Janzs bone marrow and genetically modified them in a lab. Meanwhile, he underwent chemotherapy to clear out the remaining faulty cells in his bone marrow before he got the newly modified ones. The chemo gave Janz sores in his mouth so painful that he couldnt eat. He lost his head full of pale-blond hair.
But Janz, then a 20-year-old from Alberta, Canada, had signed up for this because he knew that cystinosis was slowly killing him. The mutated gene behind this disease was causing toxic crystals of a molecule called cystine to build up everywhere in his body. He threw up constantly as a kid. Visible crystals accumulated in his eyes. And his kidneys were now failing. Cystinosis patients live, on average, to 28.5 years old.
Fortunately, the experimental gene therapy seemed to work; Janz began to feel better. His hair grew back in a stubble, but to his shock, it came in a different color: dark, almost black. In the two and half years since, his hair has settled into a dark blond, which is still markedly different from the almost white blond of before. My girlfriend actually said the other day that she feels like shes dating a different person, Janz told me.
Of all the things the experimental gene therapy was expected to altersuch as the severity of his cystinosis symptomshair color was not one of them. That was very surprising, Stephanie Cherqui, a stem-cell scientist at UC San Diego and the principal investigator of the gene-therapy trial, told me. But as she and her colleagues dug into the literature on the disease, they found that darker hair wasnt a sign of something going awry; instead it might be a very visible sign of the gene therapy working.
Doctors had observed years ago that cystinosis patients tend to be paler than their families. Manythough certainly not allhave blond hair and pale skin. One study in mice found that the gene thats mutated in cystinosis patients normally plays a role in the production of the dark-brown pigment melanin. Janz had always been a bit self-conscious about how pale he was. His whole family is pretty pale, Janz said. But I'm, like, a whole different paleor I was. The hair change, as far as hes concerned, was a nice surprise.
But how did genetically modifying his blood cells change his hair color? While the mutation that causes cystinosis affects virtually every cell in his body, gene therapy did not change the DNA of every cell in his body, only a tiny fraction of them. Scientists chose to genetically tweak blood stem cells because they have a special ability: Some eventually become white blood cells, which travel to all different parts of the body, Jeffrey Medin, who studies gene therapy at the Medical College of Wisconsin, told me. White blood cells normally go into all our different tissues and organs to patrol for pathogens.
Janzs new white blood cells were genetically modified to express the gene that is mutated in cystinosis, called CTNS. Once they traveled to his eyes, skin, and gut, the white blood cells began pumping out the missing protein encoded by the gene. Cells in the area began taking up the protein and clearing away long-accumulated cystine crystals. In Janz, the anti-cystine proteins from his modified blood cells must have reached the hair follicles in his skin. There, they cleared out the excess cystine that was blocking normal melanin production, and his hair got darker. The same phenomenon has played out in other people: So far in the gene-therapy trial, four of the five patientsall of whom are whitehave gotten darker hair. (The fifth patients hair is just starting to grow back post-therapy.) The investigators have since added hair biopsies to the trial in order to track the color changes in a more systematic fashion.
The sudden hair-color changes were surprising to Cherqui and her colleagues, but they are consistent with the role of the cystinosis gene in hair pigments, says Robert Ballotti, a melanin researcher at the French National Institute of Health and Medical Research. But he has also found that pigmentation and cystinosis can interact in unexpected ways. Not all people with cystinosis are pale, and in particular, Black patients tend not to have skin or hair that is any lighter. Maybe there is not a strict correlation between the gravity of the disease and pigmentation, Ballotti says.
Hair color is one way in which patients in the clinical trial are teaching scientists about the full scope of the CTNS gene, which is still not fully understood. Cherqui had helped discover the gene, as a graduate student more than 20 years ago, and her research has hinted at other functions for it in cell growth and survival, too. More and more, we understand that there are many functions of the protein that we didn't know, she said.
Thats why patients on the standard treatment, a drug called cysteamine, still get sicker and die of their disease, Cherqui said. Removing cystine is not enough. It doesnt help that cysteamine has some pretty nasty side effects: It causes stomach pain, nausea, and diarrhea. When Janz was very young, he needed a stomach tube to get the medication around the clock. Cysteamine also has a rotten, fishlike smell. I had a lot of difficult times as a younger kid, says Jacob Seachord, another patient in the trial whose hair went from blond to brown. I smelled really bad from medication, so I didn't make a lot of friends.
Gene therapy actually replaces the missing protein, theoretically filling in all of its functions, known and unknown. All five patients in the gene-therapy trial have gone off their oral cysteamine, and preliminary data show they now have fewer cystine crystals in their eyes, skin, and gut. Their vision has gotten slightly better, too. But improvements in kidney function are more elusive. Seachord had a kidney transplant before the gene therapy and is doing well. Janz had advanced kidney disease before the trial, and he will need a kidney transplant in a few months.
For adults with cystinosis, Cherqui said, it may be too late for gene therapy to help their kidneys. They have already accumulated a lifetime of kidney damage from cystine. Gene therapy cant reverse the damage thats been done, but we can correct it going forward, Medin said. We can stop progression. In diseases like cystinosis, patients may have to get gene therapy at a young age, probably before 10, Cherqui said. If it works, a future kid who has cystinosis might be cured through gene therapypreventing them from needing a lifetime of cysteamine or a kidney transplant. And it just might change their hair color, too.
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An Experimental Gene Therapy Changed His DNAAnd His Hair Color - The Atlantic
Alaunos Therapeutics : and the National Cancer Institute Extend Cooperative Research and Development Agreement for Development of Personalized TCR-T…
Alaunos Therapeutics and the National Cancer Institute Extend Cooperative Research and Development Agreement for Development of Personalized TCR-T Cell Therapies To 2025
NCI will lead the Company's personalized TCR-T cell therapy program using the Company's proprietary non-viral Sleeping Beauty technology
HOUSTON, June 27, 2022 - Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company, today announced that the Company has extended its Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), an institute of the National Institutes of Health, using the Alaunos Sleeping Beauty technology through January 2025.
Under the terms of the CRADA, the NCI will work to generate proof of concept utilizing the Company's proprietary non-viralSleeping Beauty technology for personalized TCR-T cell therapy. In this setting, T-cell receptors (TCRs) that react to the patient's tumor will be identified from the patient and used to generate a TCR-T cell therapy. This approach could potentially apply to a wide range of solid tumor cancer patients. Alaunos believes that the non-viralSleeping Beauty technology could rapidly and cost effectively produce safe and potent TCR-T cell therapies without the complexity of gene editing or viral approaches. Research conducted under the CRADA will be led by Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI's Center for Cancer Research.
"We are privileged to extend the productive collaboration with Dr. Rosenberg, a cell therapy pioneer. Dr. Rosenberg and the NCI are working to develop personalized cancer therapies using our novel TCR-T cell platform," commented Kevin S. Boyle, Sr., Chief Executive Officer of Alaunos. "Our collaboration reinforces our commitment to improving the lives of cancer patients with solid tumors. We look forward to continuing our collaborating with Dr. Rosenberg and his team to generate proof of concept in this personalized TCR-T approach."
Drew Deniger, Ph.D., Vice President, Research & Development at Alaunos added, "Having worked alongside Dr. Rosenberg for many years, I am confident that his team at the NCI will be successful in developing personalized TCR-T therapies using our non-viralSleeping Beauty technology. As the world's experts in Sleeping Beauty, we believe that our non-viral means of adding the TCR to T cells is well suited for a personalized approach, with potential to further increase the addressable population for TCR-T therapies."
About Alaunos Therapeutics
Alaunos is a clinical-stage oncology-focused cell therapy company, focused on developing T-cell receptor (TCR) therapies based on its proprietary, non-viralSleeping Beauty gene transfer technology and its TCR library targeting shared tumor-specific hotspot mutations in key oncogenic genes including KRAS, TP53 and EGFR. The Company has clinical and strategic collaborations with The University of Texas MD Anderson Cancer Center and the National Cancer Institute. For more information, please visit http://www.alaunos.com.
Forward-Looking Statements Disclaimer
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," "believes" or other words or terms of similar meaning. These statements include, but are not limited to, statements regarding the Company's business and strategic plans, the anticipated outcome of preclinical and clinical studies by the Company or its third-party collaborators, the Company's cash runway, and the timing of the Company's research and development programs, including the anticipated dates for filing INDs, enrolling and dosing patients in and the expected timing for announcing preclinical data and results from the Company's clinical trials. Although the management team of Alaunos believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Alaunos, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include, among other things, changes in the Company's operating plans that may impact its cash expenditures; the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Alaunos' product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Alaunos' intellectual property rights; and competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Alaunos, including those risks and uncertainties listed in the most recent periodic report filed by Alaunos with the Securities and Exchange Commission. Alaunos is providing this information as of the date of this press release, and Alaunos does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events, or any other reason.
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Alaunos Therapeutics Inc. published this content on 27 June 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 27 June 2022 12:14:02 UTC.
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Technical analysis trends ALAUNOS THERAPEUTICS, INC.
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Alaunos Therapeutics : and the National Cancer Institute Extend Cooperative Research and Development Agreement for Development of Personalized TCR-T...
Carbon Biosciences Launches with $38 Million Series A Financing to Advance Novel Gene Therapy Platform and Pipeline – Business Wire
BOSTON--(BUSINESS WIRE)--Carbon Biosciences (Carbon), a Longwood Fund founded biotech company and emerging leader in the development of novel parvovirus-derived gene therapies, today announced a $38 million Series A financing led by Agent Capital. Agent is joined by Longwood Fund, Astellas Venture Management LLC, the Cystic Fibrosis Foundation, Solasta Ventures, University of Tokyo Innovation Platform (UTokyoIPC), and Camford Capital. Carbon is harnessing novel parvovirus vectors that can deliver larger gene therapy payloads with enhanced tissue specificity and with minimal neutralizing immunity. The company plans to use the Series A funding to advance the development of Carbons programs for genetic diseases, initially building on the groundbreaking research of scientific co-founders John F. Engelhardt, Ph.D., Director, Center for Gene Therapy at the University of Iowa, and Robert M. Kotin, Ph.D., Professor of Microbiology and Physiological Systems at the University of Massachusetts Chan Medical School.
Carbons platform has the potential to expand the role of gene therapy in treating some of the worlds most devastating and difficult to treat diseases, said Joel Schneider, Ph.D., President and CEO of Carbon Biosciences. Our vision is to enable a new generation of genetic medicines with differentiated vectors that have the potential to address the immunological, targeting and payload limitations inherent in current viral and non-viral delivery technologies. As the first jointly funded program launched under a collaborative agreement between the Cystic Fibrosis Foundation and Longwood Fund, we are motivated by the potential impact our research may have in significantly improving care for cystic fibrosis patients.
Identifying vectors that can effectively deliver therapeutics to target tissues, such as the lung, has been a major challenge in realizing the full potential of gene therapy, said Geeta Vemuri, Ph.D., Managing Partner and Founder of Agent Capital. We believe that Carbon's proprietary platform can address this challenge by leveraging novel vectors from the broader parvovirus family to deliver optimal payloads to specific tissues. Carbons technology will enable a diversified pipeline with potential applications across the wide range of tissues impacted in many unaddressed diseases.
Carbons novel platform addresses key challenges with AAV and non-viral based therapies. Our lead program is the first gene therapy program demonstrating tissue tropism to the lung with the capacity to deliver the full length CFTR gene and an appropriate promotor, said John F. Engelhardt, Ph.D., the Roy J. Carver Chair in Molecular Medicine, and Director, Center for Gene Therapy at the University of Iowa. Preliminary pre-clinical data as well as studies on human populations suggest wide applicability of our lead clinical candidate and the potential to re-dose patients.
Carbons scientific co-founders and scientific advisory board members are among the most experienced thought leaders in the gene therapy field. In addition to Kotin and Engelhardt, Carbons co-founders are David Steinberg, who served as founding CEO (Longwood Fund); Jianming Qiu, Ph.D., (University of Kansas); Ziying Yan, Ph.D., (University of Iowa); Sebastian Aguirre, Ph.D., (Carbon Biosciences); and Lucy Liu, Ph.D., (Longwood Fund). Carbons Scientific Advisory Board consists of John Engelhardt; Jay Chiorini Ph.D., Senior Investigator at NIH National Institute of Dental and Craniofacial Research; Beverly Davidson, Ph.D., Chief Scientific Strategy Officer at Childrens Hospital of Philadelphia; and Adrian Thrasher, Ph.D., MBBS, FRCP, MRCP, Professor at University College London (UCL), Great Ormond Street Institute of Child Health, Head of Infection and Inflammation and Director, Clinical Gene Therapy GMP Facility at UCL.
In conjunction with the financing, Joel Schneider, Ph.D, joins as President and CEO. Chen Schor, Adicet Bio President and CEO, joins as Board Chair. David Steinberg, Geeta Vemuri, Robert Kotin and Derek Yoon, President and CEO of Solasta Ventures, join the Board of Directors.
About Carbon Biosciences
Carbon Biosciences is expanding the therapeutic potential of gene therapy through its proprietary platform which leverages novel parvoviruses that have been pressure tested by nature to target specific tissues and carry a larger cargo with minimal neutralizing immunity and the potential to re-dose. Founded by Longwood Fund and gene therapy pioneers, John F. Engelhardt, Ph.D., and Robert M. Kotin, Ph.D., Carbon is expanding the gene therapy toolbox for the treatment of the worlds most devastating and difficult to treat diseases. For more information, please visit our website http://www.carbonbio.com and follow us on LinkedIn.
About Longwood Fund
Longwood Fund is a venture capital firm dedicated to creating and investing in novel healthcare companies that develop important treatments to help patients while targeting significant value for investors. The Longwood team has a long history of successfully launching and building important life science companies while providing operational leadership and strategic guidance. Collectively, the Partners at Longwood Fund have co-founded 24 companies with over 20 launched or marketed drugs and therapies, as well as over two dozen clinical stage assets, all focused on helping patients in need. Companies founded by Longwood Fund, or its principals prior to the founding of the Firm, as lead investor and CEO/CBO include Vertex, Acceleron, Momenta, Alnylam, Sirtris, Vor, TScan, Pyxis Oncology, Immunitas, Be Biopharma, ImmuneID, Tome Bio, Photys, and Carbon Biosciences. For more information, visit http://www.longwoodfund.com.
About Agent Capital
Agent Capital is an international life sciences investment firm that supports disruptive healthcare companies focusing on novel, differentiated therapeutics and treatments that address unmet patient needs. Agent Capital aligns with scientists, entrepreneurs, and other investors to develop the next generation of healthcare innovations, leverages their industry expertise and successful track record to source premier deals, accelerate value, and drive successful exits. Their first fund invested in 15 portfolio companies, the majority of which have executed collaborations with major pharmaceutical companies and successfully raised additional capital in the private or public markets. For more information, please visit Agent Capital's website at http://www.agentcapital.com.
Originally posted here:
Carbon Biosciences Launches with $38 Million Series A Financing to Advance Novel Gene Therapy Platform and Pipeline - Business Wire
The biggest US biotech investments in May 2022 – Labiotech.eu
The DNA sequencing newcomer Ultima Genomics won the crown for the biggest U.S private biotech investment in May 2022. Other top fundraising firms are developing gene and cell therapies in addition to cultured leather and omics-based diagnostics.
The headliner private biotech investment in the US in May 2022 went to the Californian firm Ultima Genomics. The company exited stealth mode with $600 million to fund the development of high-throughput sequencing technology. The aim is to bring down the cost of sequencing the whole genome to just $100 compared to the roughly $1,000 it costs with current technology.
Ultima Genomics is upping the scale of its sequencing platform by blending a range of advances in fluidics, sequencing chemistry and machine learning. The firm is applying its sequencing technology to multiple types of omics approaches, including whole-genome sequencing, single-cell, and methylation sequencing.
The second biggest private biotech investment in the U.S. went to Kriya Therapeutics, which is based in California and North Carolina. The $270 million Series C round will advance Kriyas preclinical-stage gene therapy pipeline for the treatment of a range of ophthalmological and rare genetic conditions.
The biggest private investment raised by an industrial biotechnology firm in North America in May 2022 was an $82 million Series C round by Vestaron. Based in North Carolinas Research Triangle Park, the firm is developing peptides to protect crops from insect pests. Unlike some chemical pesticides in current use, Vestarons products are designed to be non-toxic for humans and other vertebrates that arent the direct target of the pesticide.
North Americas biggest biotech Series A round was closed by the San Francisco company Terremoto Biosciences. The company will use the winnings to fund the development of a type of small molecule that binds to proteins in a different way than traditional drugs do. This allows the company to put in the crosshairs proteins that were previously considered undruggable.
The Series A runner up in May 2022 was VitroLabs in California, which raised $47.4 million to fund the development of cultured leather. Cultured leather involves farming animal cells to produce leather, rather than using the whole animal. Like the field of cultured meat, the intention is to reduce the land, energy and water requirements of manufacturing animal-derived products.
In the seed round division, the champion was San Diego-based Pleno Inc., which took home $15 million. The company is developing a multi-omic platform to accelerate clinical diagnostics and research. By using a new type of signal-processing technology, the firm claims it can obtain much more information from polymerase chain reaction (PCR) tests and next-generation sequencing technology than from current methods.
Another notable seed round went to the New York-based Oviva Therapeutics, Inc. The firm raised $11.5 million to fund research into the aging process in women. This research focuses on slowing aging in the ovaries, which could lead to a longer lifespan in general.
One major biotech investment in the U.S. in May didnt make this list because it was of a new investment firm called Enavate Sciences. Enavate was launched by the healthcare investment firm Patient Square Capital with a $300 million boost and aims to support drug developers with vital cash as they bring novel medicines to the market.
Looking forward to June, were already seeing some huge investments, with the frontrunner so far being a $625 million Series D round by the biomanufacturing heavyweight National Resilience, Inc.
Cover image via Elena Resko
Read the rest here:
The biggest US biotech investments in May 2022 - Labiotech.eu
Intesa Sanpaolo S p A : increases its support of university research under the scope of the NRRP – The Bank partners the Universities of Bologna,…
PRESS RELEASE
INTESA SANPAOLO INCREASES ITS SUPPORT
OF UNIVERSITY RESEARCH UNDER THE SCOPE OF THE NRRP
THE BANK PARTNERS THE UNIVERSITIES OF BOLOGNA, MILAN,
NAPLES AND PADUA TO SPEED UP PROJECTS ON
BIG DATA, SUSTAINABLE MOBILITY, AGRITECH AND GENE
THERAPIES
Milan, 27 June 2022 - Intesa Sanpaolo intends to strengthen its support of university research by taking part in four National Centres for chain research as founding member of the four Foundations linked to important universities, such as the Bologna Technopole together with the Italian National Institute for Nuclear Physics (INFN), the Polytechnic University of Milan, the University of Naples Federico II and the University of Padua, which will develop new technologies for the sustainable, green digital transition, in line with the European Union Research strategic agenda and the fourth Mission of the NRRP, which finances the new centres with 1.6 billion.
Intesa Sanpaolo consolidates its commitment with the aim of speeding up the activity in synergy with the public sector, to promote the innovation and dissemination of technologies, fostering the sharing of routes and projects between universities, research entities, enterprises and start-ups. With this intervention, Intesa Sanpaolo, the only banking group of the founding members, will be able to take part together with Research Centres and Universities, in the development of projects, being an active partner in the development of technological transfer models to local enterprises and helping determine the strategic guidelines of research, as well as contributing towards the pursuit of the NRRP.
More specifically, the four Centres are as follows:
Thanks to the four new Centres, the Bologna Technopole, the Polytechnic University of Milan and the universities of Naples and Padua will act as hubs connecting with numerous other universities throughout Italy, involving an ever greater number of sites, researchers and enterprises with a view to carrying out advanced research on the topics, respectively, of big data, sustainable mobility, agritech and gene therapy.
The new Centres in fact operate through a Hub&Spoke model, in which the hubs, comprising the centres operating in synergy with some of Italy's most important universities and various private companies, will coordinate and manage the activities of the spokes, represented by entities and other local universities aiming to aggregate public and private subjects interested in making a contribution towards research into specific topics across the country.
Intesa Sanpaolo will be operating directly in 16 spokes, making the professionalism and competences present in the various bank structures involved in these research areas available, as well as technological resources useful to pursuing the objectives.
The main goal is to increase the growth potential of the Italian economic system, through a significant increase in investments in research and development and fostering the technological transfer between universities and enterprises. The new national centres will be able to help reduce or eliminate Italy's delays in terms of innovation: according to an analysis carried out by the Intesa Sanpaolo Research Department, despite the progress made in recent years, Italy is still a long way behind other European countries, and Germany in particular, in terms of spending in research and development. In this context, the interaction is facilitated between the academic and production worlds, allowing for an improvement in the number of manufacturing businesses that collaborate with universities (8%), considerably below the German figure (18%).
The Intesa Sanpaolo Group has always supported investments by enterprises in research and development, both through the management of public incentives for R&D&I and through direct financing for research and innovation. Over time, Intesa Sanpaolo has assessed approximately 2,000 R&D projects with direct financing in excess of 2.5 billion for SMEs, large enterprises and start-ups: this is flanked by the offer of technological, industrial and financial consultancy services for participation in European Research and Innovation programmes and Intellectual Property. Intesa Sanpaolo also supports innovative SMEs and start-ups with a market share respectively of 50% and 30%, also through specific initiatives, believing them to be one of the main vehicles of innovation for enterprises.
The new initiative integrates with the Group's global strategy, which, thanks to dedicated structures like the Innovation Centre and the newly-established CENTAI laboratory for advanced research in Artificial Intelligence, is present in the main Italian accelerators and acts as a nationally-important interlocutor in regard to research and innovation.
Carlo Messina, CEO of Intesa Sanpaolo: " Research centres are a great opportunity for both the Italian university research system and the business world and it is essential we support their
development. Our adhesion to this initiative is a concrete response by the Bank, which makes new resources available to speed up a structural transition in our country towards new models, closely correlated with the NRRP. We aim to offer a new boost to combine private initiative and public decision-makers, like universities, and help generate a culture of knowledge and innovation, to the
benefit of the entire social and economic system".
Ferruccio Resta, Chairman of CRUI: "The investments and measures implemented by the NR RP acknowledge a central role played by research and advanced training in developing a country that seeks to, and indeed must, grow innovatively. They provide a clear interpretation of the relationship between university and enterprise, which is increasingly close on the essential topics guaranteeing competitiveness in both the public and private sectors. The extensive, shared participation of Intesa Sanpaolo, leading Italian bank, shows the value of a tool (the National Centres), which if used properly can assure a significant change that will be completed well beyond 2026".
Press information
Intesa Sanpaolo
Media Relations Territorial Bank and Local Media stampa@intesasanpaolo.comhttps://group.intesasanpaolo.com/en/newsroom
About Intesa Sanpaolo
Intesa Sanpaolo is Italy's leading banking group - serving families, businesses and the real economy - with a significant international presence. Intesa Sanpaolo's distinctive business model makes it a European leader in Wealth Management, Protection & Advisory, highly focused on digital and fintech. An efficient and resilient Bank, it benefits from its wholly- owned product factories in asset management and insurance. The Group's strong ESG commitment includes providing 115 billion in impact lending by 2025 to communiti es and for the green transition, and 500 million i n contributions to support people most in need, positioning Intesa Sanpaolo as a world leader in terms of social impact. Intesa Sanpaolo is committed to Net Zero by 2030 for its own emissions and by 2050 for its loan and investment portfolios. An engaged patron of Italian culture, Intesa Sanpaolo has created its own network of museums, the Gallerie d'Italia , to host the bank's artistic heritage and as a venue for prestigious cultural projects.
News: group.intesasanpaolo.com/en/newsroom/news
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LinkedIn: linkedin.com/company/intesa-sanpaolo
Disclaimer
Intesa Sanpaolo S.p.A. published this content on 27 June 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 27 June 2022 10:43:02 UTC.
Publicnow 2022
Technical analysis trends INTESA SANPAOLO S.P.A.
Income Statement Evolution
Increasing Healthcare Expenditure to Drive Sales of Reprocessed Medical Devices at 14.9% CAGR through 2028: Fact.MR Survey – BioSpace
North America Is Expected To Account For Around 40% Of The Global Reprocessed Medical Devices Market Share By The End Of 2028
Increasing focus on healthcare in developing nations with less spending potential will see major adoption of reprocessed medical devices through 2028, says a Fact.MR analyst
Fact.MR A Market Research and Competitive Intelligence Provider: The reprocessed medical devices market stood at a valuation of US$ 2.05 Bn in 2020, and is projected to surge to US$ 5.9 Bn by the end of 2028.
Increasing healthcare expenditure owing to the rising patient pool is expected to bolster demand for reprocessed medical devices as healthcare institutions focus on lowering operational costs. Increasing geriatric population, favorable regulatory policies, and rising focus on healthcare are anticipated to be other trends propelling reprocessed medical devices market potential over the coming years.
Demand for reprocessed medical devices is anticipated to be high in developing economies where healthcare spending potential is less.
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Key reprocessed medical device suppliers are focusing on launching new and innovative products to advance medical device reprocessing procedures and increase revenue potential.
Key Takeaways from Market Study
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Winning Strategy
Reprocessed medical device manufacturers are focusing on mergers and acquisitions to expand their product portfolios and increase their sales. Favorable regulatory policies are also expected to promote reprocessed medical device suppliers to advance their sales potential and increase revenue.
Reprocessed medical device companies are investing in the development of better reprocessing facilities to meet increasing demand. Favorable government initiatives to boost the adoption of reusable medical devices are also expected to be capitalized on by key reprocessed medical devices market players over the forecast period.
Competitive Landscape:
Key reprocessed medical device market players are focusing on mergers and acquisitions to increase their business scope across the world and increase sales.
Key Segments Covered in Reprocessed Medical Devices Industry Research
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More Valuable Insights on Offer
Fact.MR, in its new offering, presents an unbiased analysis of the global reprocessed medical devices market, presenting historical demand data (2017-2020) and forecast statistics for the period of 2021-2028.
The study divulges essential insights on the market on the basis of device type (cardiovascular devices, general surgery devices, laparoscopic devices, orthopedic external fixation devices, gastroenterology biopsy forceps) and across six major regions (North America, Latin America, Europe, East Asia, South Asia & Oceania, and the Middle East & Africa).
Fact.MRs Domain Knowledge in Healthcare
Our healthcare consulting team guides organizations at each step of their business strategy by helping you understand how the latest influencers account for operational and strategic transformation in the healthcare sector. Our expertise in recognizing the challenges and trends impacting the global healthcare industry provides indispensable insights and support - encasing a strategic perspective that helps you identify critical issues and devise appropriate solutions.
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Increasing Healthcare Expenditure to Drive Sales of Reprocessed Medical Devices at 14.9% CAGR through 2028: Fact.MR Survey - BioSpace
CRISPR Technology Market: Rise in Demand for Gene Therapeutics to Drive the Global Market – BioSpace
Wilmington, Delaware, United States: CRISPR is a genome-editing tool. It is used by researchers/health care professionals to alter DNA sequences and modify gene function.
CRISPR is also used to correct genetic defects, treat human diseases, and yield better crop varieties
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CRISPR stands for Clusters of Regularly Interspaced Short Palindromic Repeats. The CRISPR-Cas9 system consists of two key molecules: Cas9 and guide RNA, which introduces a change or mutation into the DNA at the desired position.
Cas9 is an enzyme that acts as a pair of molecular scissors and can cut the two strands of DNA at a specific location in the genome so that bits of DNA can then be added or removed
The guide RNA (also known as gRNA) is a small piece of pre-designed RNA sequence (about 20 bases long) located within a longer RNA scaffold. The scaffold part binds to DNA and the pre-designed sequence guides Cas9 to the right part of the genome.
This makes sure that the Cas9 enzyme cuts at the right point in the genome. High orthogonality, versatility, and efficiency of CRISPR technology make it a preferred genome editing technology.
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Key Drivers, Restraints, and Opportunities of Global CRISPR Technology Market
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In March 2020, researchers at New York genome center developed a new CRISPR screening technology to target RNA, including RNA of novel viruses such as COVID-19. In November 2019, researchers at ETH Zurich, Switzerland, swapped CAS9 enzyme for CAS 12a, which allowed the researchers to edit genes in 25 target sites.
North America to Dominate Global CRISPR Technology Market
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Key Players of Global CRISPR Technology Market
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CRISPR Technology Market: Rise in Demand for Gene Therapeutics to Drive the Global Market - BioSpace