Archive for the ‘Gene Therapy Research’ Category

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

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

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

Some of the key highlights of Glioblastoma Multiforme Pipeline Report

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

Some of the Glioblastoma Multiforme Pipeline Therapies

Regorafenib: Bayer Healthcare

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

CYNK 001: Celularity

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

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

Upcoming Glioblastoma Multiforme Therapies

Scope of Glioblastoma Multiforme Pipeline Therapeutics report

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

Table of Contents

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

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

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

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

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

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

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

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

About Graphite Bio

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

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

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

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

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

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

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

Scope of the Report

Key Topics Covered:

1. Research Methodology and Executive Summary

1.1 Research Methodology

1.2 Executive Summary

2. Strategic Recommendations

3. Gene Therapy Market: Product Outlook

4. Global Gene Therapy Market: Sizing and Forecast

4.1 Market Size, By Value, Year 2015-2025

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

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

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

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

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

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

5.2 Competitive Scenario of Global Gene Therapy Market: By Application

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

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

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

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

6. Global Gene Therapy Market: Regional Analysis

6.1 Competitive Scenario of Global Gene Therapy Market: By Region

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

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

7.2 North America Gene Therapy Market - Prominent Companies

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

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

7.5 North America Gene Therapy Market: Country Analysis

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

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

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

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

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

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

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

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

10. Global Gene Therapy Market Dynamics

10.1 Drivers

10.2 Restraints

10.3 Trends

11. Market Attractiveness

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

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

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

12. Competitive Landscape

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

12.2 Product Pipeline of Leading Gene Therapy Companies

12.3 Market Share Analysis

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

13.1 Voyager Therapeutics

13.2 Novartis AG

13.3 Spark Therapeutics Inc.

13.4 MoldMed S.P.A.

13.5 Orchard Therapeutics PLC

13.6 Alnylam Pharmaceuticals Inc.

13.7 AnGes Inc.

13.8 Akcea Therapeutics

13.9 BlueBird Bio Inc.

13.10 Sarepta Therapeutics

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

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

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

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

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

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

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

About DB-020

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

About Decibel Therapeutics, Inc.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Contact the author Uttara Choudhury at [emailprotected]

Follow her on Twitter: @UttaraProactive

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Orgenesis leads the charge at an exciting time for cell and gene therapy - Proactive Investors USA & Canada

Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act.

But a key analyst covering biopharma believes that theres a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, hes pointing to the 2 leaders in the field as the most likely players to come out ahead way, way ahead.

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Regeneron teams with gene therapy pioneer James Wilson, adapting its Covid-19 antibody cocktail to an AAV-based nasal spray - Endpoints News

Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act.

But a key analyst covering biopharma believes that theres a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, hes pointing to the 2 leaders in the field as the most likely players to come out ahead way, way ahead.

Unlock this story instantly and join 94,500+ biopharma pros reading Endpoints daily and it's free.

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UPDATED: Bayer continues its cell and gene therapy push, enveloping different projects under one strategic roof while hunting new deals - Endpoints...

4D Molecular Therapeutics raised $75 million in June to get several gene therapy programs into and through the clinic. Now, its adding a trio of executives to spearhead its work in heart, eye and lung diseases as it looks to shepherd treatments in those focus areas forward.

Robert Fishman, M.D. becomes 4Ds chief medical officer and therapeutic area head for pulmonology. He joins from Xoc Pharmaceuticals, where as chief medical officer he led phase 1 development for programs in Parkinsons disease and migraine. Before that, he headed clinical development at InterMune, overseeing the pivotal trial of Esbriet, an idiopathic pulmonary fibrosis drug now marketed by Roche.

Accelerate Biologics, Gene and Cell Therapy Product Development partnering with GenScript ProBio

GenScript ProBio is the bio-pharmaceutical CDMO segment of the worlds leading biotech company GenScript, proactively providing end-to-end service from drug discovery to commercialization with professional solutions and efficient processes to accelerate drug development for customers.

RELATED: Restoring eyesight with genetically engineered stem cells

Raphael Schiffmann, M.D., signs on as senior vice president and therapeutic area head for 4Ds cardiology stable. He was previously director of the Institute of Metabolic Disease at the Baylor Research Institute and the lead investigator of the developmental and metabolic neurology branch at the NIHs National Institute of Neurological Disorders and Stroke.

Robert Kim, M.D., joins 4D as a senior vice president and clinical therapeutic area head of ophthalmology. Hes held multiple chief medical officer roles at ViewPoint Therapeutics, Apellis Pharma and Vision Medicines, and earlier in his career worked in ophthalmology at GlaxoSmithKline, Genentech and Novartis.

The three executives arrive six months after 4D topped up its coffers with a $75 million series C round. The capital, which came two years after a $90 million B round, was earmarked to push three programs into the clinic, including two that are partnered with Roche.

Those programs include 4D-310, a treatment for Fabry disease in which patients cells accumulate a type of fat called globotriaosylceramide, and 4D-125, a treatment for the eye disease X-linked retinitis pigmentosa. Roche has the exclusive right to develop and commercialize the latter. Roche has licensed the third prospect, 4D-110, a treatment for a type of vision loss called choroideremia.

RELATED: 4D raises $90M to move gene therapies into clinical testing with AstraZeneca and Roche

The funds will also bankroll the development of 4Ds preclinical pipeline, including IND-enabling studies for 4D-710, a program in cystic fibrosis, and other candidates for neuromuscular diseases and ophthalmology.

With the addition of Robert Fishman, Raphael Schiffmann and Robert Kim to our clinical R&D leadership team, 4DMT gains not only extensive experience in clinical development and translational medicine, but also unique and specific experience within each of the initial 4DMT therapeutic areas," said 4D CEO David Kim, M.D., in a statement.

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4D hires a trio of area heads as it ramps up its gene therapy pipeline - FierceBiotech

London, 2nd December 2020 Cell and Gene Therapy Catapult (CGT Catapult) today announces the launch of the Advanced Therapies Skills Training Network (ATSTN). It is designed to reach more people than ever before to propel them towards career opportunities in the advanced therapy and vaccine manufacturing industry by assessing their transferrable skills and providing access to training.

Driven by industry and coordinated by the CGT Catapult, the ATSTN delivers a platform consisting of dedicated online resources and national centres to upskill as well as the opportunity for people to identify how they can enter this industry. This is a significant contribution to ensuring continuous growth of the UK advanced therapies and vaccine manufacturing industry, developing the nations health resilience by creating opportunities for current and new skilled personnel.

The ATSTN initiative is funded by 4.7 million awarded from the Department for Business, Energy & Industrial Strategy (BEIS) through UK Research and Innovation (UKRI) to help grow the sector by creating economic opportunities for new jobs and industry driven learning. With the industry workforce expected to double to more than 6,000 by 2024, this investment is continuing the Governments commitment to expanding the UK expertise in advanced therapies.

Business Secretary Alok Sharma, said:

It is vital thatwe upskillourworkforce todevelopand manufacture vaccinesand treatmentsat scale,ensuring the advanced therapy and vaccine manufacturing sectors are stronger and more resilient in the future.

Backed with almost 5 million government investment, this first-of-its-kind skills network will equip people at all stages of their careersto pursueopportunities in advanced medicines and vaccine manufacturing. This will help to create highly skilled jobsacross the countrywhile putting the UK in the strongest position to respond to future pandemics.

Kate Bingham, Chair of the Vaccines Taskforce said:

One of the tasks of the governmentsVaccines Taskforceis to ensure the UK has the capabilities to respond to future health crises,and this includes scaling up our ability to manufacture safe and effective vaccines and treatments.

By upskilling and expanding the UKs vaccine manufacturing industry, this new skills network will help to ensure the UK has the capacity to produce successful vaccines at scale, including for coronavirus, helping to protect the UK population for generations to come.

Matthew Durdy, CEO of Cell and Gene Therapy Catapult commented:

The ATSTN initiative reiterates our commitment to upskill new and existing members of the advanced therapy workforce, building on the success and impact of the ATAC apprenticeship programme. Not only is it rewarding to work so closely with industry to deliver on this need, but it is also extremely motivating for all involved to think that this initiative is going to be the start of so many promising careers and additional job opportunities. We look forward to seeing the success stories from users of this ground-breaking platform

The ATSTN has been designed in collaboration with industry to address its needs to upskill current professionals as well as to leverage skills from other sectors, opening opportunities for people to join the cell and gene therapy industry from different ones. ATSTN users will be able to access learning materials, whether remotely or on-site, from a wealth of expertise across the entire industry, while tailoring their personal learning experience to their own needs.

There are three different components to the ATSTN platform: Online Training Platform, National Training Centres and Career Converter. These components allow users, whether they come from inside or outside the industry, to curate job matches based on their current skillset and identify what training they need to progress in that field.

The National Training Centres, that will provide on-site training, are to be announced later in December.

The ATSTN platform can be accessed from http://www.atskillstrainingnetwork.org.uk

- END-

Notes to editor:

About the ATSTN learning platform features

The networks Career Converter tool is a great place to start for people looking to enter the industry by identifying what skills they already have. Once they have built a comprehensive skills profile, the tool will match the candidate with the advanced therapy roles most suited to them as well as a breakdown of the training they would need.

The Online Training Platform is a place for candidates to enrol onto courses so they can start filling the gaps on their skills profile right away. Once they are logged in, they will be able to browse the online course catalogue and schedule their learning at their own pace. The platform, developed in collaboration with industry, can help build skills in areas specific to industry sectors as well as general training into leadership and people skills.

List of organisations that have been involved to date with the development of ATSTN

Achilles Therapeutics, Adaptimmune, Agylia, Allergan Biologics, Anthony Nolan Cell and Gene Therapy Services, AskBio, Autolus, Biogelx, Cancer Research UK, Cellular Therapies Facility Newcastle, Cell and Gene Therapy Catapult, Charles River, Cobra Biologics, CPI, Enginuity, EvOx Therapeutics, eXmoor, Freeline, Fujifilm, GOSH, GSK, Gyroscope, Immetacyte, Intertek, Medicines Discovery Catapult, John Goldman Centre for Cellular Therapy Imperial College, Merck SAFC, National Horizons Centre, NHSBT, HakoBio designed by OUAT!, Oxford BioMedica, Oxford University Clinical Biomanufacturing Facility, Pall Corporation, Phion Therapeutics, Replimmune, RoslinCT, Symbiosis, UKRI, VIMIC.

About Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

For further information please contact

FTI Consulting for the CGT Catapult:

Michael Trace +44 (0) 203 319 5674 / michael.trace@fticonsulting.com

George Kendrick + 44 (0) 203 727 1411 / george.kendrick@fticonsulting.com

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UK set to take advanced therapies and vaccine manufacturing industry to the next level: the Advanced Therapy Skills Training Network launches today -...

Dec. 2, 2020 06:00 UTC

LONDON--(BUSINESS WIRE)-- Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced that Jennifer Cook, David Fellows and Rene Gal have been appointed to the Gyroscope Board of Directors, effective immediately.

I am very pleased to welcome Jennifer, David, and Rene, three extremely accomplished executives with extensive biopharmaceutical backgrounds, to our board of directors, said Khurem Farooq, Chief Executive Officer. We look forward to their valuable contributions and unique perspectives as we continue our work to develop gene therapy beyond rare disease with the goal of delivering new medicines to people with retinal diseases.

Ms. Cook was most recently the Chief Executive Officer of GRAIL, a healthcare company focused on the early detection of cancer. Prior to joining GRAIL, she held a number of leadership positions during her 25-year tenure with Roche/Genentech, including leading Roche Pharmaceuticals European commercial business, Global Clinical Operations, U.S. and Global Product Portfolio Management, the U.S. Immunology and Ophthalmology Business Unit and Market Development. She currently serves on the boards of directors of Denali Therapeutics, BridgeBio Pharma and Ambys Medicines. Ms. Cook holds a B.A. in human biology and an M.S. in biology from Stanford University, as well as an MBA from the Haas School of Business at the University of California, Berkeley.

Mr. Fellows was most recently the Chief Executive Officer of Nightstar Therapeutics, a retinal gene therapy company acquired by Biogen in 2019. Prior to joining Nightstar, he was the Vice President of Johnson & Johnsons Vision Care Franchise where he led the global marketing, new product and licensing activities. Before joining Johnson & Johnson, Mr. Fellows held leadership positions at Allergan, Inc., for 25 years where he served primarily in sales and marketing in a number of capacities, including Regional President, Corporate Vice President, and Senior Vice President across North America, Europe and Asia. He currently serves as the chairman of the board for Oxular Limited and is a board member of the Glaucoma Foundation. Mr. Fellows holds a B.A. in psychology from Butler University.

Ms. Gal currently serves as the Executive Vice President and Chief Financial Officer of Jazz Pharmaceuticals, a global biopharmaceutical company. Prior to joining Jazz, she served as the Chief Financial Officer of GRAIL. Before joining GRAIL, Ms. Gal served as the Senior Vice President and Chief Financial Officer of Theravance Biopharma, where she led the companys spin-out transaction from Innoviva, Inc. Ms. Gal has also served in global treasury, pharmaceutical sales and corporate strategy/business development at Eli Lilly and Company. Prior to joining Eli Lilly, she spent seven years in the energy industry in positions focused on corporate finance, project finance and mergers and acquisitions. She currently serves on the board of directors of Gossamer Bio where she chairs its audit committee. Ms. Gal holds a B.S. in mathematics from Vanderbilt University and an MBA from Columbia Business School.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing gene therapy beyond rare disease to treat a leading cause of blindness, dry age-related macular degeneration (AMD). Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and Phase II clinical trials called EXPLORE and HORIZON.

Syncona Ltd, our lead investor, helped us create a leading retinal gene therapy company combining discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201201006277/en/

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Gyroscope Therapeutics Announces Appointments of Industry Veterans in Gene Therapy, Ophthalmology and Biopharmaceuticals to Its Board of Directors -...

Researchers inserted genes into mouse eye cells to protect and restore the optic nerve (red fibers in microscope image of healthy mouse retina, above).

By Kelly ServickDec. 2, 2020 , 5:30 PM

Do old and damaged cells remember what it was like to be young? Thats the suggestion of new study, in which scientists reprogrammed neurons in mouse eyes to make them more resistant to damage and able to regrow after injurylike the cells of younger mice. The study suggests that hallmarks of aging, and possibly the keys to reversing it, lie in the epigenome, the proteins and other compounds that decorate DNA and influence what genes are turned on or off.

The idea that aging cells hold a memory of their young epigenome is very provocative, says Maximina Yun, a regenerative biologist at the Dresden University of Technology who was not involved in the work. The new study supports that [idea], but by no means proves it, she adds. If researchers can replicate these results in other animals and explain their mechanism, she says, the work could lead to treatments in humans for age-related disease in the eye and beyond.

Epigenetic factors influence our metabolism, our susceptibility to various diseases, and even the way emotional trauma is passed through generations. Molecular biologist David Sinclair of Harvard Medical School, who has long been on the hunt for antiaging strategies, has also looked for signs of aging in the epigenome.

The big question was, is there a reset button? he says. Would cells know how to become younger and healthier?

In the new study, Sinclair and his collaborators aimed to rejuvenate cells by inserting genes that encode reprogramming factors,which regulate gene expressionthe reading of DNA to make proteins. The team chose three of the four factors scientists have used for more than 10 years to turn adult cells into induced pluripotent stem cells, which resemble the cells of an early embryo. (Exposing animals to all four factors can cause tumors.)

The team focused specifically on neurons at the back of the eye called retinal ganglion cells. These cells relay information from light-sensitive photoreceptors to the brain using long tendrillike structures called axons, which make up the optic nerve. Theres a stark divide between youth and age in these cells: An embryonic or newborn mouse can regenerate the optic nerve if it gets severed, but that ability vanishes with time.

To test whether their treatment could bring back some of that resilience, Sinclair and colleagues crushed the optic nerves of mice using forceps and injected a harmless virus into the eye carrying the genes for the three reprogramming factors. The injection prevented some damaged retinal ganglion cells from dying and even prompted some to grow new axons reaching back to the brain, the team reports today in Nature.

When the researchers looked at methylation patternsthe DNA location of chemical tags called methyl groups that regulate gene expressionthey found that changes caused by the injury resembled those in aging mouse cells. In certain parts of the genome, the treatment reversed those changes. The researchers also found that the benefits of the introduced genes depended on cells ability to alter their methylation patterns: Mice lacking certain enzymes necessary to remove methyl groups from DNA saw no benefit to the treatment.

Thats really something special, says Leonard Levin, a visual neuroscientist at McGill University. The experiments suggest how the famous and well-studied reprogramming factors restore cells. But big questions remain, he says: How do these factors cause methyl groups to be added or removed? How does that process help retinal ganglion cells?

Sinclairs team also tested the approach in mice with a condition meant to mimic glaucoma, a leading cause of age-related blindness in humans. In glaucoma, the optic nerve gets damaged, often by a buildup of pressure in the eye. Sinclair and his colleagues injected tiny beads into the animals eyes that prevented normal drainage and increased pressure, which damaged retinal ganglion cells.

Four weeks later, the animals visual acuity had declined by about 25%, as measured by a vision test in which mice move their heads to track the movement of vertical bars displayed on computer monitors. But after the genetic treatment, the animals gained back roughly half of their lost acuitythe first demonstration of restored vision in mice after this glaucomalike injury.

Still, the improvement in acuity was small, Levin notes. And, he says, the treated mice were in a relatively early stage of damage, not the state of near or total blindness that people experience when glaucoma goes untreated for years. So its too early to say whether this approach could benefit people who have lost much of their vision. Levin adds that there are already very good treatments for early-stage glaucoma to prevent vision loss with medicated eye drops or surgery to lower eye pressure.

In a final set of experiments, Sinclair and colleagues injected the reprogramming-factor genes into the eyes of 1-year-old healthy mice, roughly the mouse equivalent of middle-age. By this stage, the animals had visual acuity scores about 15% lower than their 5-month-old counterparts. Four weeks after treatment, older mice had similar acuity scores to younger ones. In their cells, the researchers saw patterns of DNA methylation and gene expression resembling those of younger animals.

In the three sets of experiments, Sinclair says, the cells seemed to respond to the reprogramming factors by fine-tuning their gene expression to match a youthful state. He sees that behavior as a hint that cells keep a record of their epigenetic past, even though its not clear how that record is stored. A company Sinclair cofounded, Life Biosciences, is developing treatments for diseases associated with aging, including glaucoma, and he says hes now planning to test the safety of this gene therapy approach in larger animals.

Yun says that as a strategy for reversing aging or treating disease, resetting the epigenome is a very difficult one. Reprogramming cells to an earlier state carries a risk of prompting uncontrolled growth and cancer.Future studies should test how the three factors affect other types of cells and tissues and confirm that reprogrammed cells maintain their youthful state long-term, she says. There are a lot of roads to be traveled.

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Researchers restore lost sight in mice, offering clues to reversing aging - Science Magazine

"The need for reproducible, scalable, and economical production of cell and gene therapies is creating a demand for digital bioprocessing technologies," said Nitin Naik, Global Life Sciences Vice President at Frost & Sullivan. "These technologies are critical to realize the true commercial potential of cell and gene therapies in the next two to three years and serve as a conduit to improve market access and control the total cost of therapy."

Naik added: "From a market segment perspective, while the stem cell market is lucrative, the highest growth is expected to be in gene-modified cell therapies, with a pipeline of 269 products,* followed by gene therapies, which account for 182 assets in the pipeline.* Further, although allogeneic stem cell therapies dominate the marketed product catalogs, interest in disease-modifying CAR-T therapies, which are largely autologous, is driving demand for the evolution of manufacturing technologies, models, and capacity expansion investment by CDMOs." (*as of August 2020)

To tap into the growth prospects exposed by the CGT market, companies must focus on:

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026 is the latest addition to Frost & Sullivan's Healthcare research and analyses available through the Frost & Sullivan Leadership Council, which helps organizations identify a continuous flow of growth opportunities to succeed in an unpredictable future.

About Frost & Sullivan

For six decades, Frost & Sullivan has been world-renowned for its role in helping investors, corporate leaders and governments navigate economic changes and identify disruptive technologies, Mega Trends, new business models and companies to action, resulting in a continuous flow of growth opportunities to drive future success.Contact us: Start the discussion.

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026

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Contact:Mariana Fernandez Corporate Communications P: +1 210 348 10 12 E: [emailprotected] http://ww2.frost.com

SOURCE Frost & Sullivan

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Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan - PRNewswire

Executive Summary Global Gene Therapy Market was valued at USD 1221. 84 Million in the year 2019. Escalating number of cases related to various chronic diseases including Cancer, Cardiovascular and Neurological Disorders, Alzheimers and Sickle Cell Diseases, with companies investing heavily in incorporating advanced technology supported by growing collaboration between bio-pharma companies and research institutes to advance in the field of Gene therapy, has been anticipated to infuse growth in the market for Gene Therapy during the forecast period of 2020-2025.

New York, Dec. 01, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Gene Therapy Market Analysis By Vector Type, By Application, By Region, By Country (2020 Edition): Market Insights, Covid-19 Impact, Competition and Forecast (2020-2025)" - https://www.reportlinker.com/p05993452/?utm_source=GNW

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

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

Scope of the Report The report analyses Gene Therapy market By Value. The report analyses Gene Therapy Market By Vector Type (Lentivirus, AAV, Retrovirus & Gammaretrovirus, Others). The report further assesses the Gene Therapy market By Application (Neurological Disorders, Cancer, Cardiovascular Diseases, Others). The Global Gene Therapy Market has been analysed By Region (North America, Europe, and Asia Pacific) and By Country (United States, Canada, Germany, France, United Kingdom, Italy, China, Japan, India, South Korea). The key insights of the report have been presented through the frameworks of Major Mergers & Acquisitions, Technological Innovations, and Role of Companies during COVID-19 Pandemic with and Product Pipeline of Leading Companies. Also, the attractiveness of the market has been presented By Region, Vector Type and Application. Additionally, the major opportunities, trends, drivers and challenges of the industry has been analysed in the report. The companies analysed in the report include Voyager Therapeutics, Novartis AG, Spark Therapeutics Inc., MoldMed S.P.A., Orchard Therapeutics PLC, Alnylam Pharmaceuticals Inc., AnGes Inc., Akcea Therapeutics, BlueBird Bio Inc., and Sarepta Therapeutics. The report presents the analysis of Gene Therapy market for the historical period of 2015-2019 and the forecast period of 2020-2025.

Key Target Audience Bio-Tech and Bio-Pharma Companies Hospitals, Clinics, and Healthcare Units Consulting and Advisory Firms Investment Banks and Equity FirmsRead the full report: https://www.reportlinker.com/p05993452/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Global Gene Therapy Market Analysis By Vector Type, By Application, By Region, By Country (2020 Edition): Market Insights, Covid-19 Impact,...

Janssen is seeking approval of amivantamab for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

This morning, the company submitted a Biologics License Application to the U.S. Food and Drug Administration for this indication. Amivantamab is an investigational fully-human EGFR and mesenchymal epithelial transition factor (MET) bispecific antibody that targets both adriver mutation as well as a resistance mechanism. Exon 20 insertion mutations account for at least 9% of EGFR mutations. However, there are no FDA-approved targeted therapies for patients with these mutations, which means chemotherapy remains the standard of care, Janssen said this morning.

EGFR mutations, can lead to uncontrolled cancer cell growth and division. Those mutations are some of the most common mutations in NSCLC.EGFR exon 20 insertion mutations are the third most prevalent primary EGFR mutation, but often go undetected, the company said.

The BLA submission for amivantamab is based on data from the monotherapy arm of the Phase I CHRYSALIS study, a multi-center, open-label, multi-cohort study evaluating the safety and efficacy of amivantamab as a monotherapy and in combination with lazertinib, a novel third-generation EGFR tyrosine kinase inhibitor (TKI), in adult patients with advanced NSCLC. The BLA for amivantamab marks the first regulatory submission for patients with exon 20 insertion mutations and also marks Janssens first filing for the treatment of patients with lung cancer, the company said.

Elsewhere today:

Taysha Gene Therapies Texas-based Taysha Gene Therapies, Inc. secured both Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration for TSHA-103, an AAV-9-based gene therapy in development for SLC6A1-related epilepsy, the company announced this morning.

RA Session II, founder and chief executive officer of Taysha, said the company is pleased with the designations awarded by the FDA and encouraged by the results of the ongoing research.

SLC6A1 epilepsy is an autosomal dominant genetic disorder characterized by the loss of function of one copy of theSLC6A1gene, with clinical manifestations of seizures, epilepsy, language impairment and intellectual disability. Steven Gray, chief scientific advisor to the company and an associate professor in the Department of Pediatrics at UT Southwestern, said haploinsufficiency in theSLC6A1gene has been identified as a cause of genetic epilepsy. However, as of yet, there are no approved disease-modifying therapies for this indication, he said. Gray touted the FDA designations and said they highlight the importance of developing a treatment for patients.

BioAge Labs California-based BioAge Labs raised $90 million in an oversubscribed Series C financing found. Funds will be used to support the development of BioAges platform that maps key pathways that drive human aging, as well as its pipeline of treatments that targets those pathways, BioAge CEO Kristen Fortney said in a statement. BioAge anticipates taking its first platform-derived therapies, BGE-117 and BGE-175 into clinical trials in the first half of 2021.

The financing round was co-led by Andreessen Horowitz and serial entrepreneur, Elad Gil. New investors include Kaiser Foundation Hospitals, AARP Foundation and Phi-X Capital. Current investors in the company, including Caffeinated Capital, Redpoint Ventures, PEAR Ventures, AME Cloud Ventures and Felicis Ventures, also participated.

Kinnate Biopharma Bay Area-based Kinnate Biopharma announced the pricing of a $20 per share price for its initial public offering of 12,000,000 shares of common stock. Gross proceeds are expected to be $240 million. The stock will be available for trading starting today on the Nasdaq exchange under the ticker symbol KNTE. Kinnate Biopharma is focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers. Proceeds from the IPO will be used to fund clinical trials, for R&D and other general corporate purposes, the company said.

Valneva Frances Valneva SE said it will accelerate pediatric development of its Lyme vaccine candidate, VLA15, in collaboration with Pfizer. The company intends to begin dosing in the first quarter of 2021. The Phase II VLA15-221 study will include approximately 600 healthy participants between the ages of 5 and 65. If greenlit by health authorities, the study will be the first clinical trial of VLA15 to enroll a pediatric population aged 5-17 years and will compare the three-dose vaccination schedule Month 0-2-6 with a reduced two-dose schedule of Month 0-6. This study will complement the two ongoing Phase II studies VLA15-201 and VLA15-202, both of which posted positive data earlier this year. All three Phase II trials are anticipated to support a Phase III pivotal efficacy trial including all main target populations for the Lyme vaccine candidate starting in 2022.

Most read today on BioSpace:

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Daily Rundown: Who's Gunning For and Gaining Approval and Other Industry News - BioSpace

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTX), a next-generation gene therapy company with an industry-leading RNA targeting technology platform, announces today the unveiling of the AAVidTM capsid discovery platform and results from its first AAV5 variant library in a non-human primate selection campaign.

The AAVidTM capsid discovery platform uses non-random mutational fitness to create massive capsid libraries of billions of unique AAV variants for direct-to-NHP in vivo biological selection. By combining cutting-edge DNA synthesis, advanced synthetic biology, next-generation sequence barcoding and machine learning algorithms, ShapeTX generates industry-leading library size and diversity to enable the development of best-in-class human therapeutics.

Wildtype first-generation AAVs are enabling the recent advances in gene therapy, but they have been plagued by toxicities in the clinic due in part to a lack of tissue specificity, resulting in the need for high doses. Our AAVidTM platform solves the issue by creating novel capsid variants with specific tissue-tropism, said Francois Vigneault Ph.D., President and CEO at ShapeTX. Weve stayed quiet for the past three years while developing a superior AAV platform technology and are excited to announce that we have best-in-class AAV variants in hand. Today, we are announcing our novel liver-tropic AAV5 variants stay tuned for more to come.

David J. Huss, Ph.D., Vice President and Head of Research added, The vast structural space for exploration at the AAV capsid/target cell interface necessitates enormous library size and diversity, which until now, has only been probed with capsid library sizes in the tens of thousands to millions. At ShapeTX, we set out to create a superior AAV capsid discovery platform with library sizes in the billions of unique variants, thereby maximizing the opportunity for novel virus/target cell interactions. Dr. Huss presented the details of the platform at the 2nd RNA Editing Summit on Dec. 2, 2020.

About Shape Therapeutics, Inc

Shape Therapeutics is a biotechnology company developing next-generation RNA-targeted therapies to treat the worlds most challenging diseases. The ShapeTX technology platform includes RNAskip, a proprietary suppressor tRNA technology that enables premature stop codon readthrough; RNAfixTM, a precision RNA editing technology using endogenous Adenosine Deaminase Acting on RNA (ADAR); and AAVidTM, a next-generation engineered adeno-associated virus (AAV) platform producing highly specific, tissue-tropic AAVs. The power of the ShapeTX platforms resides in redirecting the cellular machinery already present in our cells, thereby bypassing the risks of immunogenicity and DNA damage seen with other contemporary editing technologies. ShapeTX is committed to data-driven scientific advancement, passionate people and a mission of providing life-long cures to patients. Shape Life!

Originally posted here:
Shape Therapeutics Unveils AAVid Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery...

Fort Collins, Colorado Global Cancer Gene Therapy Market report on in-depth research, sales estimates, and growth forecast details by segments, regions and research scope, historical data, key players, and growth value.

The Cancer Gene Therapy Market 2020 analysis provides a basic summary of the trade along with definitions, classifications, uses, and trade chain structure. Global Cancer Gene Therapy market study is provided for the international markets, along with development trends, competitive landscape analysis, and key regions development status. Development policies and plans are mentioned in the same way as production processes and value structures are analyzed. This report also provides information on import/export consumption, supply and demand, costs, prices, sales, and gross margins.

Global Cancer Gene Therapy Market industry valued approximately USD 0.2 billion in 2016 is anticipated to grow with a healthy growth rate of more than 35.1% over the forecast period 2017-2025.

Download Exclusive Sample PDF along with few company profiles @ https://reportsglobe.com/download-sample/?rid=5801

The Major Players Profiled in this Report include

Impact of Covid-19 on this Market:

Cancer Gene Therapy Market report analyses the impact of Coronavirus (COVID-19) on the Cancer Gene Therapy industry. Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Cancer Gene Therapy market in 2020.

The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; emergency declared in many countries; massive slowing of the supply chain; stock market unpredictability; falling business assurance, growing panic among the population, and uncertainty about future.

COVID-19 can affect the global economy in 3 main ways: by directly affecting production and demand, by creating supply chain and market disturbance, and by its financial impact on firms and financial markets.

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Market Segments and Sub-segments Covered in the Report are as per below:

By End-User:

By Therapy:

This Market Study covers the Cancer Gene Therapy Market Size across segments. It aims at estimating the market size and the growth potential of the market across segments by component, data type, deployment type, organization size, vertical, and region. This Cancer Gene Therapy study also includes an in-depth competitive analysis of the key market players, along with their company profiles, key observations related to product and business offerings, recent developments, and key market strategies.

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Finally, the Cancer Gene Therapy Market Report is a credible source of market research that will accelerate your business exponentially. The report gives the most important regional framework conditions, economic situations with item value, advantage, limit, production, supply, demand, market development rate and number, etc. Cancer Gene Therapy Industry Report Also includes a new SWOT review task, speculative test research, and corporate return on investment research.

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Cancer Gene Therapy Market Size, Opportunities, Dynamic, Outlook and Forecast To 2027 - Cheshire Media

Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.

With 61 codons specifying 20 amino acids, some can be encoded by more than one codon and it is often presumed that it does not matter which one a gene uses. When I first studied genetics, some books I read taught that mutations between such alternative codons (eg, GGA->GGC, both giving glycine) were called synonymous mutations, while others referred to them as silent mutations. However, are synonymous mutations really silent meaning they are identical in terms of fitness and function? Although they may specify the same amino acid, does that mean they are all the same?

Figure 1: Intronless GFP transgene expression is higher for variants of GFP with higher GC content at synonymous sites5

Perhaps one of the biggest surprises over recent years has been the discovery that versions of the same gene, differing only at synonymous sites, can not only have different properties, but effects that are not modest.1-5 For example, two versions of green fluorescent protein (GFP) differing only at synonymous sites can have orders of magnitude differences in their expression level.4 We similarly recently discovered that for an intronless transgene to express in human cell lines it needs to be GC rich, which can be achieved by altering the synonymous sites,5 as seen in Figure 1. It is no accident, we suggest, that the well-expressed endogenous intronless genes in humans (such as histones) are all GC rich and that our functional retrogenes tend to be richer in GC content than their parental genes.

The realisation that synonymous sites matter has clear relevance to the design of transgenes or other artificial genes, be these for experiments, gene therapy, protein production (eg, in bacteria) or for vaccine design. In the case of vaccines, we might wish to modulate a viral protein to be effectively expressed in human cells to illicit a strong and robust immune response.6 Conversely to the design of attenuated vaccines, we seek to produce a tuned down version of the virus that can function but is weak.7

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered. One approach is mass randomisation try many alternatives and see what works.4,8,9 In principle this is fine, but this approach requires many randomisations, which is still technically difficult for long attenuated viruses. An alternative strategy that we have been exploring is to let nature tell us; we can apply tools and ideas from population genetics to better understand what natural selection favours and disfavours and in turn to estimate the strength of selection.

it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus

Estimation of the strength of selection is possible from knowledge of the site frequency spectrum, (ie, how common variants are) from which we can infer the distribution of fitness effects (DFE). If a site is under strong purifying selection, then mutations may occur in the population but these are rapidly eliminated, so variants are always rare. By contrast, if they are selectively neutral, we expect some variants to be quite common. We recently applied this methodology to show that synonymous mutations in human genes that disrupt exonic splice enhancer motifs are often under strong selection and affect many synonymous sites in our genes.10 This has implications for both diagnostics and for transgene design for gene therapy, as we often remove introns in heterologous genes, so freeing up these residues from their role in specifying exons ceases.11

The same DFE methodology cannot easily be applied to viruses, as the methods assume free recombination (ie, we assume one mutation does not impact the fate of others in the same genome). However, other population genetical tools can still be applied. Recently, we examined SARS-CoV-2 and identified the profile of mutations that we see at four-fold degenerate sites.12 From this profile we could estimate what the synonymous site composition would be, assuming that the only forces are mutational biases and neutral evolution (ie, no selection). We observed that in this genome there is a strikingly strong C->U mutation bias and a G->U one. In the raw data this is not so obvious as G and C are quite rare. However, the mutability of the sites per occurrence of the site reveals the underlying patterns.

Figure 2: The rate of mutational flux from one dinucleotide to another in the coding sequence of SARS-CoV-2. The direction of flux is indicated by the indentation of the connecting links: the inner layer represents flux out while the outermost layer represents flux into the node. The frequency of the flux exchange is represented by the width of any given link where it meets the outer axis. Dinucleotide nodes are coloured according to their GC-content. Hence, it is evident that there is high flux away from GC-rich dinucleotides whereas AU-rich dinucleotides are largely conserved.12

With knowledge of the mutational bias we then asked what the equilibrium frequency of the four nucleotides would be using four simultaneous equations. This is the nucleotide content at which for every mutation changing a particular base there is an equal and opposite one creating the same base somewhere else in the genome, ensuring overall unchanged nucleotide content. Given the strong C->U and G->U mutational biases, it is no surprise that the equilibrium content is very U rich (we estimate equilibrium U content should be about 65 percent). However, while the four-fold sites are indeed U rich, they are not that U rich, being closer to 50 percent. A clue as to why the mutation bias is so skewed to generating U comes from analysis of equilibrium UU content: UU residues are predicted to be very common, with CU residues being particularly mutable generating UU (Figure 2) this is expected due to human APOBEC proteins attacking and mutating/editing the virus.13

One probable explanation for this difference between predicted and observed nucleotide content is selection against U content. There may be many U residues appearing in the population, but many are pushed out of the population owing to purification selection, ie, because of the deleterious effects of the mutations. That such selection is happening in the SARS-CoV-2 genome is also clear from the sequence data. We estimate that for every 10 mutations that appear in the sequence databases, another six are lost because of selection prior to genome sequencing. Indeed, UU content is about a quarter of that predicted (Figure 3).

Figure 3: The predicted (under neutral mutational equilibrium) and observed dinucleotide content of SARS-CoV-2. Note the very high predicted levels of UU given the strong mutational flux to UU residues (see Figure 2) and the net underrepresentation in actual sequence.9

This leaves two problems: why is selection operating on SARS-CoV-2 and what can we do with this information? In some cases, we have a good idea as to why: many mutations to U at codon sites generate stop codons. However, we have observed that U destabilises the transcripts and is associated with lower-reported transcript levels;12 a full explanation of the causes of selection on nucleotide content therefore requires manipulation of the sequences.

The second question, what to do with this information, is perhaps more urgent. It has previously been noted that nucleotide content manipulation is a viable means to attenuate viruses.7 Currently there are three groups investigating this route to make a vaccine for SARS-CoV-2: Indian Immunologicals Ltd/Griffith University, Codagenix/Serum Institute of India and Acbadem Labmed Health Services/Mehmet Ali Aydinlar University. In prior attempts, attention has been paid to CpG levels and UpA levels (which we find to be correlated between SARS genes and between different viruses).12 CpGs attract the attention of zinc antiviral protein (ZAP) and UpA attracts an RNAase L. Not surprisingly, some viruses, including SARS-CoV-2, therefore have low levels of both dinucleotide pairs given the levels of the underlying nucleotides.

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered

In the past, attenuation strategies have focused on modulating synonymous sites to increase CpG and UpA, making the virus more visible to antiviral proteins.14 We in turn suggest a general strategy to utilise this method and to increase U content as well.12 Given the evidence that selection on the virus is to reduce U content, while our antiviral proteins are mutating it to increase U content, it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus. This is an unusual circumstance in which we predict that we should build in more of the already most common synonymous site nucleotides (U in this case) to degrade the virus. More generally, it is assumed that the most used codons are those that tend to increase the fitness of the organism. In the face of such a severe mutation bias, however, this simpler logic no longer holds.

Laurence D Hurst is Professor of Evolutionary Genetics and Director of the Milner Centre for Evolution at the University of Bath. He is currently also the President of the Genetics Society. He completed his D.Phil in Oxford, after which he won a research fellowship and then moved to Cambridge University as a Royal Society Research Fellow. While on the fellowship he assumed his current Chair at Bath University. In 2015 he was elected a Fellow of the Academy of Medical Sciences and a Fellow of the Royal Society. He is a recipient of the Genetics Society Medal and the Scientific Medal of the Zoological Society of London.

Related topicsDisease research, DNA, Gene Therapy, Genetic analysis, Genomics, Protein, Proteogenomics, Proteomics, Research & Development, RNAs, Vaccine

See more here:
Tweaking synonymous sites for gene therapy and vaccines - Drug Target Review

FREDERICK, Md., Dec. 02, 2020 (GLOBE NEWSWIRE) -- RoosterBio, Inc, a leading supplier of innovative cellular therapy bioprocess tools and scalable manufacturing solutions for regenerative medicine, today announced it entered into an exclusive agency agreement with Sartorius Korea Biotech, a subsidiary of the Sartorius Group, a leading international partner of the biopharmaceutical and life science research sectors. This strategic partnership combines Sartorius proven expertise in providing high-caliber bioprocess testing, technical and customer services to the local SouthKorean market, as well as its global reach, and RoosterBios advanced platform of RUO and cGMP-grade human mesenchymal stem/stromal cell (hMSC) working cell banks, optimized paired media, and hMSC bioprocess systems. This winning combination sets the stage for accelerating the development of new, effective medical treatments and expands RoosterBios footprint into Asia, especially in South Korea.

South Korea is a preeminent innovator in hMSCs, and the South Korean biotech ecosystem has been flourishing according to every measure of growth. One of the first MSC therapies was developed in Korea, clearing an entirely new path to treat previously incurable diseases, said RoosterBio CEO Margot Connor. We are delighted to be working with Sartorius Korea Biotech in our global effort to industrialize the supply chain for next-gen treatments, and we are committed to accelerating the commercialization of cell- and gene-based therapies. We believe the talented team at Sartorius is uniquely suited to provide our mutual customers with scalable hMSC systems to expedite their cell-based therapeutic programs.

Under the terms of the agreement, Sartorius Korea Biotech will engage with its customers as an exclusive agent for RoosterBio to facilitate sales operations and increase the market potential for RoosterBio products on an exclusive basis in South Korea.

RoosterBios commercialized product systems standardize hMSC manufacturing to remove years of time and millions of dollars from the timeline of traditional mesenchymal cell therapeutic product development and clinical translation efforts, said Duck Sang Kim, Managing Director of Sartorius Korea Biotech. We look forward to introducing this synergistic biotechnology relationship to South Korea.

About RoosterBio, Inc

RoosterBio, Inc. is a privately held cell manufacturing platform technology company focused on accelerating the development of a sustainable Regenerative Medicine industry, one customer at a time. RoosterBio's products are high-volume, affordable, and well-characterized adult human mesenchymal stem/stromal cells (hMSCs) paired with highly engineered media systems. RoosterBio has simplified and standardized how living cells are purchased, expanded, and used in development, leading to marked time and costs savings for customers. RoosterBio's innovative products are ushering in a new era of productivity and standardization into the field. Visit http://www.roosterbio.com.

About Sartorius Korea Biotech, Ltd.

Sartorius Korea Biotech based in Pangyo, South Korea, addresses the evolving needs of the biomanufacturing industry to produce biotech medications and vaccines safely and efficiently. The company is a subsidiary of the Sartorius Group, a leading international partner of life science research and the biopharmaceutical industry, and offers a diversified business portfolio reflecting the products and services of the two Group divisions, Bioprocess Solutions and Lab Products & Services. Sartorius Korea Biotech provides pharmaceutical and laboratory equipment, validation service, training, engineering and consulting, and KOLAS calibration, among other products and services.

Visit http://www.sartorius.com

Contact: Carrie Zhang, Director of Marketing, RoosterBioczhang@roosterbio.comAndre Hofmann, Head of Public Relations, Corporate Communications, Sartoriusandre.hofmann@sartorius.com

See the article here:
RoosterBio Collaborates with Sartorius to Expand Cell and Gene Therapy Manufacturing Platform Technology into South Korea - GlobeNewswire

Blood vessels are supposed to act like trees, pumping in oxygen tissues need to grow and immune cells required to clear out pathogens. But in tumors, the forest can go a bit haywire. Vessels grow prodigiously and bulge and twist at abrupt points, making it difficult to even tell whats a vein and whats an artery. It starts to look less like a forest and more like a gnarled root floor. A disorganized labyrinth, one oncologist has called it.

For cancer, chaos is a virtue. That gnarled root floor insulates solid tumors from immune cells and, in recent years, has flustered drug developers best attempts at developing therapies meant to rev up the immune system and direct it toward the tumors.

Researchers at the University of Pennsylvania, however, think they may have stumbled onto a solution, a way of whipping the blood vessels back into proper shape. If it works, experts say, it could pave the way for CAR-T treatments that attack solid tumors and potentially improve the effectiveness for more traditional approaches, such as radiation and chemotherapy.

Its a really novel and potentially important approach, Patrick Wen, a neuro-oncologist at Dana-Farber who was not involved in the work, told Endpoints News. They really did good work. This is a very different way of improving immunotherapy.

Yi Fan, a radiation oncologist and neurosurgeon at Penns School of Medicine, has been working for the last few years to understand why the labyrinth appears in the first place. Researchers had previously circled in on the so-called growth factors that stimulate blood vessel formation. Attempts to block these factors, though, disappointed; Avastin, an antibody against the factor VEGF, became a blockbuster but has continually failed to improve survival on a range of malignancies.

Scientists would have to go more fundamental. In a pair of 2018 papers, Fan showed that part of the problem is a process called endothelial cell transformation. Cells lining the blood vessels around the tumor acquire stem cell-like properties that allow them to reproduce and expand rapidly, as stem cells do.

Theres a genetic reprogramming, Fan told Endpoints. Theyll become really aggressive.

But how did that reprogramming happen? If Fan could pin down the pathway, he figured he could then devise a way to block it. He started knocking out kinases the cellular engines that can drive epigenetic change, or reprogramming one by one in endothelial cells isolated from patients with an aggressive brain cancer called glioblastoma. Out of 518, 35 prevented transformation and one did so particularly well: PAK4.

Then they injected tumors into mice, some who had PAK4 and some who had the kinase genetically removed: Eighty percent of the mice who had PAK4 removed lived for 60 days, while all of the wild-type mice died within 40. Fans team also showed that T cells infiltrated the tumors more easily in the PAK4-less mice.

It was a fortuitous finding: Drug companies had developed several PAK inhibitors a decade ago, when kinase inhibitors were the flashiest thing in pharma. Many had been abandoned, but Karyopharm had recently brought a PAK4 blocker into Phase I.

To see whether drug developers could exploit this finding, Fan and his team removed T cells from mice and developed a CAR-T therapy to attack the tumors.

They gave mice three different regimens. The CAR-T therapy on its own failed to reduce tumor size, apparently unable to reach through the vessels. The Karyopharm drug also had little effect on its own. But combined, they managed to reduce tumor size by 80% after five days. They published the results in Nature Cancer this week.

It is a really eye-opening result, Fan said. I think we see something really dramatic.

That, of course, is just in mice, but Fan already has strong supporting evidence for PAK4s role in cancer. Last December, while Fan was still completing his experiment, Nature Cancer published a paper from Antoni Ribas UCLA lab suggesting that PAK4 inhibitors can help T cells infiltrate around various solid tumors. They showed that the same Karyopharm inhibitor could boost the effects of PD-1 inhibitors in mice, allowing activated T cells to better reach tumors.

That work has already translated into the clinic; weeks after it came out, Karyopharm added an arm to their Phase I study of the drug that will look at the PAK4 inhibitor in combination with the PD-1 blocker Opdivo.

Ribas said that Fans work is compelling and helps confirm the role of PAK4, but he said a CAR-T therapy would face a much longer path to the clinic. Its simply much easier to combine an approved drug with an experimental one than to devise a new CAR-T therapy, mix it with the unapproved inhibitor (and all the other things, such as bone marrow-clearing chemotherapy, CAR-T recipients receive) and then deduce what effect each is having.

It will a take a while, Ribas told Endpoints. But I hope this is right and its developed clinically.

There are also other unresolved obstacles for CAR-T in solid tumors, Wen said. Developers still struggle to find targets that wont also send the super-charged T cells after healthy tissue. And tangled blood vessels are just one of several mechanisms tumors have of defending themselves. They can, for example, turn tumor-eating immune cells into tumor-defending ones.

Still, Wen said, in the short term, the approach offered a path toward boosting the efficacy of radiation, chemotherapy and other small molecule drugs. Although Fan focused on glioblastoma, researchers agreed PAK4 likely plays the same vessel-warping role in many other solid tumors.

Theres a lot of things you could look at, he said.

In a January review, Jessica Fessler and Thomas Gajewski at the University of Chicago said Ribas paper pointed towards a path for improving PD-1 and overcoming resistance in some tumors. But they also raised questions about the Karyopharm drug, noting that it hits other proteins besides PAK4. That could mean other mechanisms are also at play and that the drug could affect other tissues in humans.

Ribas agreed that Karyopharms drug might not be the perfect molecule but said others could be on their way. He serves as a scientific advisor to Arcus, the Terry Rosen startup that is now working on developing its own PAK4 inhibitor.

If they can develop a very selective PAK4 inhibitor, he said, it may be a more direct way of testing the role of PAK4.

Tests with that drug, in turn, could help clear up a biological mystery that emerged out of Fans and Ribas papers. Although both investigators zeroed in on PAK4, each of them suggested very different mechanisms by which PAK4 kept immune cells out of the tumor. Ribas suggested it directly suppresses T cells, while Fan found it led to those transformations inside the blood vessels near the tumor.

Kinases are versatile proteins and both researchers said its possible that PAK4 is doing both. Its also possible, they said, that one is more important than the other, or simply that one of them is just wrong.

When you start with completely new biology, its hard to get it right the first time, Ribas said.

See the original post here:
Penn researchers find a way through the labyrinth keeping CAR-T from solid tumors - Endpoints News

A little more than 2 years after Mercks Roger Perlmutter signed off on a deal that would pay Dragonfly up to $695 million for each drug program it picked off for solid tumors, the pharma giant is stepping up with their first opt-in.

We dont know exactly how much this deal costs Merck in the upfront, or which immunotherapy theyre getting, but its a major step forward for Bill Haneys Waltham, MA-based biotech, which built its TriNKET technology platform with the help of Tyler Jacks, an MIT professor, HHMI investigator and director of the David H. Koch Institute for Integrative Cancer Research as well as Berkeleys David Raulet, whose background as an expert in NK cells and tumor immunology helped spotlight some of the big ideas Dragonfly is pursuing.

This latest pact marks the latest in a flurry of BD deals for the pharma giant, just one last step before Perlmutter hangs it up as head of R&D and passes the reins to Dean Li. John Carroll

A little over a year since announcing its Series A, gene therapy biotech ViGeneron has entered into a new deal.

The German company is partnering with WuXi Advanced Therapies, a contract testing, development and manufacturing organization under WuXi AppTec based out of Philadelphia, to ramp up production of ViGenerons lead candidate VG901 for ophthalmic disorders. VG901s current target is for retinitis pigmentosa, also known as rod cone dystrophy, a degenerative eye disease that causes severe vision impairment as early as childhood.

Manufacturing for the candidate should begin before the year is out, ViGeneron said in a statement. The company added that the program came out of its proprietary vgAAV vector platform, which allows for better transduction of retinal cells as well as a less invasive treatment administration.

There is currently no cure for the disease, though there are some methods that can help manage symptoms like the use of low vision aids and portable lighting. Patients often experience worsening peripheral vision and trouble seeing at night. Max Gelman

Lentiviral vector manufacturer iVexSol has raised $13 million in Series A financing, bringing the total haul to $15.2 million from Casdin Capital and BioLife Solutions and a third undisclosed lead investor.

Founded on the promise to change the way this critical raw material is made using next-generation manufacturing tech, the company said it can produce LVVs at significantly greater quantities than traditional transient transfection processes. The companys name is short for intelligent vector solutions.

Much like adeno-associated viral vectors, or AAV, these delivery vehicles are crucial for cell and gene therapies such as CAR-T, iVexSol added, and their shortage means developers often have to wait 12 to 18 months for production slots.

Details on exactly how it plans to revolutionize the space are scant, but CEO Rod Rietze and CSO Mike Greene both bring technical experience from shops like Novartis and Pfizer.

Its new funding will help establish a facility in Lexington, MA housing stable LVV producer cell line master banks and commercial-grade LVV. Amber Tong

Continue reading here:
News briefing: Merck's Roger Perlmutter buys his first solid tumor TriNKET from Dragonfly; ViGeneron to expand production of eye gene therapy -...

Gene therapy has shown promise for the first time to help treatbowelcancerthat has spread to the liver.

Adelaide researchers showed the novel approach, which uses a modified virus to infect the liver, was able to shrink tumours in mice.

"We're very excited by these results," Dr Susan Woods, one of the investigators in the study that was published in the Gastroenterology journal, said.

Further testing will be carried out to see whether the therapy could work for othercancers that travel to the liver such as tumours of the breast, lung and pancreas.

The modified virus exclusively targets the liver and introduces a copy of a gene that instructs the organ to make more cells called fibroblasts which are known to be good and slowcancergrowth.

The team from SAHMRI and the University of Adelaide have been investigating why normal cells that surround thecancerare good while others are corrupted and promote tumour growth.

"Inbowelcancer, we know that patients with the poorest prognosis have a lot of these corrupted or bad tumour supporting fibroblasts," Dr Susan Woods said.

This type of gene therapy that uses a modified virus to enter the liver is currently being used on patients with blood disorders.

"This is the first sign that we could use this to treatcancerthat has spread to the liver," Dr Woods said.

Bowelcancersurvivor Hannah Devereux is heartened by the research and said there needs to be more treatment options for people who are diagnosed with the disease late, when it has already spread.

Hannah was only 34 when she was diagnosed withbowelcancer, soon after her second child was born.

"Had the baby, he was 10 days old, and they found two tumours. My world came crashing down," she said.

Hannah had complained about digestive symptoms during the pregnancy.

"The doctor just thought it was pregnancy related," she said.

Hannah required intensive treatment for a year including six months of chemotherapy, radiotherapy and two major surgeries.

She has now reached the five-yearcancer-free milestone and is the ambassador of the Jodi Lee Foundation to preventbowelcancer.

Bowelcanceraffects more than 15,000 Australians each year and fewer than 50 per cent of cases are detected early.

More than 100 Australians each week die frombowelcancer.

More:
New gene therapy could help treat bowel cancer that reaches the liver - 9News

~ Strong Presence at ASH Featuring Five Presentations, Including Late-BreakingOral Presentation on HOPE-B Pivotal Trial ~

~ uniQure to Host Investor Webcast Tuesday, December 8, 2020 at 5:00 p.m. ET ~

LEXINGTON, Mass. and AMSTERDAM, Nov. 30, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs,today announced that Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, will present clinical data from the HOPE-B pivotal trial of etranacogene dezaparvovec in hemophilia B at the virtual 62nd American Society of Hematology (ASH) Annual Meeting.

uniQure management along with Dr. Pipe will host an investor webcast on Tuesday, December 8, 2020, at 5:00 p.m. ET. To access the live webcast with presentation slides, please visit the Investor Relations section of uniQures website at http://www.uniQure.com. The webcast will be archived for 90 days. The event also may be accessed by dialing (877) 870 9135 for domestic callers and +44 020 719 283 38 for international callers. The passcode is 3164585. Please specify to the operator that you would like to join the uniQure Conference Call.

The conference abstracts are available and can be accessed through this link: ASH abstracts.

About uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to,whether we will present long-term follow-up data from our hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical trial of AMT-060, and whether we will announce top-line data from the pivotal HOPE-B study of etranacogene dezaparvovec before the end of this year. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures Quarterly Report on Form 10-Q filed onOctober 27, 2020. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:

Link:
uniQure Announces Multiple Presentations and Investor Webcast on Hemophilia B Gene Therapy Program at the 62nd American Society of Hematology (ASH)...

A new study on the CRISPR and Cas Genes market provides a detailed overview of the demands and consumptions of various products/services associated with the growth dynamics of the market. The in-depth market estimation of various opportunities in the segments is expressed in volumes and revenues during the forecast period 2020 to 2026. The insights and analytics on the CRISPR and Cas Genes market span several pages. These are covered in numerous sections, including, drivers and restraints, challenges and opportunities, regional segmentation and opportunity assessment, end-use/application prospects analysis, and competitive landscape assessment.The global revenues in CRISPR and Cas Genes market are projected to garner a CAGR of 21.2% from during 2020 to 2026.

Key stakeholders in the CRISPR and Cas Genes market including industry players, policymakers, and investors in various countries have been continuously realigning their strategies and approaches to implement them in order to tap into new opportunities. Many in recent months have overhauled their strategies to remain agile in the backdrop of worldwide disruptions caused by the COVID-19 pandemic.

Request for Sample Report @ https://www.factmr.com/connectus/sample?flag=S&rep_id=4823

The study also offers scrutiny of the changing government policies amid COVID-19 disruptions. Policymakers in developing and developed nations are framing new regulations to meet the continuing macrocosmic shocks by COVID-19 pandemic. The authors of the report have taken into account the impact analysis of the pandemic, and have elaborated on the trends that will be crucial to the upcoming competitive landscape. New entrants, as well as established players who want to emerge as leaders in the post-COVID era, are taking the impact analysis seriously.

The following insights and evaluations are worth knowing for any market participant, helping them in ascertaining the prevailing dynamics and the future trajectories of the CRISPR and Cas Genes market. They are a part of the estimations of the opportunities in various segments.

Some of the insights and market estimations that make this study unique in approach and effective in guiding stakeholders in understanding the growth dynamics. The study provides:

The report offers detailed regional segmentation of the CRISPR and Cas Genes market which includes shares of each key region in the global landscape during the forecast period. The segmentation is done as follows:

The CRISPR and Cas Genes market report offers a critical quantitative and qualitative assessment of the current and future avenues in the following product segments:

The global CRISPR and Cas Genes market report comes with the following end uses:

A detailed assessment of the growth dynamics includes opportunities and growth trends. Some of the questions pertain to these are answered in the study:

Send an Enquiry @ https://www.factmr.com/connectus/sample?flag=AE&rep_id=4823

The report on the CRISPR and Cas Genes market is unique in its approach and insights-gathering initiatives. Some of the aspects that the study highlight are:

Why Choose Fact.MR?

Fact.MR follows a multidisciplinary approach to extract information about various industries. Our analysts perform thorough primary and secondary research to gather data associated with the market. With modern industrial and digitalization tools, we provide avant-garde business ideas to our clients. We address clients living in across parts of the world with our 24/7 service availability.

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Fact.MR Report: What is the impact of Coronavirus on CRISPR and Cas Genes Market Growth? - The Cloud Tribune

New York, Nov. 27, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User, and Country" - https://www.reportlinker.com/p05989496/?utm_source=GNW However, the low success rate of cell therapies and the high cost of cell-based research is expected to restrain the market growth during the forecast period.

Cell therapy typically involves the administration of somatic cell preparations by injecting or grafting it into the patients body for the treatment of diseases or traumatic damages.The procedure is used to cure diabetes, neurological disorders, related injuries, several cancer types, bones and joints, and genetic disorders.

Continuous research and development activities have led to unique cell therapeutic instruments for the improvement of immune system and efficient treatment of genetic disorders. Various market players provide several consumables such as reagent kits and enzymes as well as devices, equipment, and software to perform various cell therapy processes.

The use of instruments is essential for handling cell therapies such as NSC, PSC, MSC, T cells, and HSC.These cell therapy products are derived from animals or human cells and thus need to be protected from contamination.

The instruments used in cell therapies help provide protection against contamination and allow scaling up of transplantation. Companies such as Hitachi Chemical Advanced Therapeutics Solutions Corning Incorporated; Thermo Fisher Scientific Inc., MiltenyiBiotec, LLC; Invetech; and Cytiva (General Electric Company) have introduced various equipment and consumables for the cell therapy procedures.

The global COVID-19 emergency has been particularly affecting the supply chain worldwide.The supply chain disruptions, along with the enormous demand for effective therapies for the treatment of COVID-19, have put the healthcare research industry in a crucial situation in the Asia Pacific region.

However, many medical companies have realized the importance of cell therapy in the treatment of COVID 19, which would raise its demand in the coming years.

The Asia Pacific cell therapy instruments market, by product, is segmented into consumables, software, equipment, and systems.The consumables segment held the largest share of the market in 2019 and is expected to register the highest CAGR during the forecast period.

On the basis of cell type, the cell therapy instruments market is segmented into animal cells and human cells. The human cells segment held a larger share of the market in 2019 and is estimated to register a higher CAGR during the forecast period.

On the basis of process, the Asia Pacific cell therapy instruments market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control.The cell processing segment held the largest share of the market in 2019 and is estimated to register the highest CAGR during the forecast period.

The Asia Pacific cell therapy instruments market, based on end user, is segmented into life science research companies, research institutes, and other end users. The life science research companies segment accounted for the largest share of the market in 2019 and is anticipated to register the highest CAGR during the forecast period.

A few of the major primary and secondary sources associated with the Asia Pacific cell therapy instruments market are National Center for Biotechnology Information (NCBI); World Health Organization (WHO); Medical Research Future Fund (MRFF); Asia-Pacific Economic Corporation (APEC); and Global Institute of Stem Cell Therapy and Research (GIOSTAR).Read the full report: https://www.reportlinker.com/p05989496/?utm_source=GNW

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Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User,...