Category REGENERATIVE MEDICINE

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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In Breakthrough Study, Researchers Find Stem Cells in the Nose That Give Rise to Olfactory Neurons

By carefully harvesting tiny samples of olfactory cleft mucosal tissue and conducting single-cell analysis, a collaborative team led by University of Miami Miller School of Medicine researchers has found compelling evidence to support olfactory neurogenesis, the ability to regrow olfactory neurons to the brain. “For decades, medical researchers have wondered whether or not neurogenesis was

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New research on a promising treatment against type 1 diabetes funded by the European Commission with 6,8 million euros

Within the framework of a European funded H2020 project, VANGUARD aims to develop a cure for type 1 diabetes available to a larger group of patients. The VANGUARD project will use advanced tissue engineering techniques to generate a bioartificial pancreas, with the aim to propose a novel cell-based treatment to type 1 diabetes that overcomes

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Korea Institute of Medical Microrobotics researchers publish stem cell delivery via microrobot for knee cartilage surgery

Korean researchers have successfully demonstrated knee cartilage regeneration by using stem cells delivered by a magnet-controlled microrobot, raising hope to broaden a non-invasive procedure for patients who need invasive knee replacement surgery. The new method can also raise stem cell delivery accuracy efficiency by up to 95 percent compared to less than 20 percent in

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Gene Editing and AI Can Build New Generation of Low-Cost Health Solutions, Bill Gates Says

Bill Gates calls for reducing health care inequities in a AAAS Annual Meeting plenary address. The number of child deaths around the world has declined by half in the past two decades, but that number could be cut even further with the help of artificial intelligence and gene editing tools, said Bill Gates at the

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