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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

Therapies 137
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Catapult Ventures invests in OVO Biomanufacturing to improve the manufacturing efficiency of viral vaccines and gene therapies

Drug Discovery Today

OVO Biomanufacturing, a spin-out from the University of Warwick and Coventry University, is developing digital solutions to improve the efficiency of viral vaccine and gene therapy manufacture. The technology can be applied to any virus that is grown to produce a vaccine or therapy.

Vaccine 100
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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

Therapies 119
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Analytical Testing Considerations for Gene Therapy Products

The Premier Consulting Blog

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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From Nanotechnology to Gene Therapy: Innovations in Drug Delivery

DrugBank

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.    A key part of gene therapy is efficiently delivering the therapeutic genetic material directly into target cells.

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New insights into the role of viral capsids in gene therapy safety

Drug Target Review

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Recent Advances in Gene Therapy

DrugBank

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. In sickle cell disease, for instance, a missense mutation in the HBB gene , encoding the beta-globin subunit of hemoglobin, leads to the production of abnormal hemoglobin S.