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FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

Drugs.com

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

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FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Results from the study that led to the FDA approval appeared in The Lancet Neurology in April 2024 with commentary. Researchers have been working on developing gene therapy for DMD for decades.

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Behind the new kind of cell therapy that just won FDA approval

BioPharma Drive: Drug Pricing

One expert views Amtagvi’s approval as a catalyst for further investment in TIL therapies, akin to how Kymriah’s 2017 clearance buoyed CAR-T treatment.

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FDA Approves First Gene Therapy, Elevidys, to Treat Duchenne Muscular Dystrophy

Drugs.com

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.

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FDA Approves Landmark Sickle Cell Gene Therapies

Drugs.com

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.

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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

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FDA Approves First Gene Therapy to Treat Duchenne Muscular Dystrophy

Drugs.com

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.