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The FDA approved delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics) in June to treat Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 who have a confirmed DMD gene mutation.
In this article, Jinwei Bio introduces their lentiviral manufacturing processes and discusses the various benefits this entails.
An update on the emerging evidence of the effects and safety of disease modifying therapies for the management of children with spinal muscular atrophy (SMA).
With increasing numbers of cell and gene therapies (CGTs) entering clinical trials, Dr Dave Li and Dr Anna Baran of KCR Consulting discuss how the biological characteristics of CGTs should be taken into account with early phase trial designs.
Press release content from Business Wire. The AP news staff was not involved in its creation. ASC Therapeutics Receives IND Clearance From the U.S. Food and Drug Administration for ASC618 Second-Generation Gene Therapy for Hemophilia A July 8, 2021 GMT MILPITAS, Calif.--(BUSINESS WIRE)--Jul 7, 2021-- ASC Therapeutics, a privately-held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic and other rare disorders today announced that the U.S. Food & Drug Administration (FDA) has cleared an Inv...