Category GENE THERAPY RARE DISEASES

Tackling the Challenges in Cell and Gene Therapy Manufacturing

The excitement about cell and gene therapies is almost tangible within the biotech and pharma industry. Over 950 companies are actively developing advanced therapies, which are expected to make exceptional improvements to peoples’ lives in the next decade. Although hopes are high, the industry still faces a number of challenges in cell and gene therapy manufacturing, mainly

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Plant-based relatives of cholesterol could help gene therapy for cystic fibrosis, other diseases

Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol, new research shows. That’s because the shape and structure of the phytosterols help the genes get where they need to be inside cells. The findings by Oregon State University researchers, published today in Nature Communications, are important because many illnesses that

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BioMarin’s Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020

If approved, 1st Gene Therapy in U.S. for the Treatment of Any Type of Hemophilia BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia

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New Institute Launched to Ensure the U.S. Healthcare System Is Ready for Gene Therapies

Industry leaders, patient advocates, researchers unite to maximize the incredible potential of transformative gene therapies The Institute for Gene Therapies (IGT) launched today with a focus on advocating for a modernize the U.S. regulatory and reimbursement framework so that gene therapies can deliver their significant potential to patients. IGT will educate stakeholders across the healthcare

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Novel techniques for mining patented gene therapies offer promising treatment options for cancers, other diseases

A team of scientists from Purdue University and institutions around the world have come together to better understand the growing number of worldwide patented innovations available for gene therapy treatment. The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases. Now, a team of scientists

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Supporting progress of cell and gene therapy industrialisation with new aseptic manufacturing course

The University of Hertfordshire and the Cell and Gene Therapy Catapult (CGT Catapult) have announced the launch of a new course specifically addressing the foreseeable skills gap in the manufacture of cell and gene therapies as they progress towards manufacturing at scale. Developed in a collaboration between the two organisations, this three-day course will provide

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New gene correction therapy for Duchenne muscular dystrophy. Gene scissors against incurable muscular disease

Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum München) have developed a gene therapy

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FDA Continues Strong Support of Innovation in Development of Gene Therapy Products

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more

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