Category GENE THERAPY RARE DISEASES

Researchers of NEI use gene therapy and a novel light-sensing protein to restore vision in mice

NIH-funded therapy will now be tested in humans A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy. The National Eye Institute, part of the National Institutes of Health, provided a Small Business Innovation Research grant to Nanoscope, LLC for development of

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Clinical Development of Gene Therapies: The First Three Decades and Counting.

In the past three decades the field of gene therapy has made remarkable progress surging from mere laboratory experiments to FDA-approved products which bring significant reduction in disease burden to patients who previously had no therapeutic options for their serious conditions. In this paper it’s reviewed the evolution of the gene therapy clinical research landscape

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362 cell and gene therapies in US pipeline, finds report

New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018. A new report from America’s Biopharmaceutical Companies has revealed that there are 362 cell and gene therapies in development in the US. Roughly a third of the therapies, 132, are potential treatments for rare diseases.  The research

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Scientists at Oregon edit genes inside the body using CRISPR for a hereditary blindness disorder, in a trial called BRILLIANCE.

A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body. The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that cause a rare condition called Leber’s congenital amaurosis 10 (LCA10).

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Scientists of Case Western Reserve University successfully test new way to deliver gene therapy

Researchers use lipids to safely deliver gene therapy to the eye, successfully holding off advance of rare, inherited eye disorder Researchers at Case Western Reserve University have used a unique method to safely deliver gene therapy to fight a rare, but irreversible, genetic eye disorder known as Stargardt disease.  By using chemically modified lipids—instead of the

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Gene therapy generates new neurons to treat Huntington’s disease

Gene therapy to treat Huntington’s disease Huntington’s disease (HD) is a rare disease characterized by abnormal chorea movement and caused by Huntingtin (Htt) gene mutation and neurodegeneration in a brain area called striatum. A research group led by Dr. Gong Chen, a former professor at Penn State University and now leading a brain repair center

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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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