Gene Therapy
Latest News
March 20th 2025
Encelto is small, semi-permeable capsule implanted in the eye that contains allogeneic retinal pigment epithelium cells genetically engineered to produce specific therapeutic proteins. It will be available in June 2025.
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Elevidys Misses Primary Endpoint, but Shows Evidence of Modifying the Course of Duchenne
October 31st 2023Sarepta Therapeutics plans to submit an application for full approval of Elevidys to treat all ages of patients with Duchenne muscular dystrophy. Company officials said the FDA is open to evaluating the application based on the totality of the evidence.
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Female Carriers of X-Linked Retinal Diseases Advocate for Gene Therapy Access
October 30th 2023Many carriers reported feelings of concern, anxiousness, and guilt for passing the X-linked inherited retinal disease to their children—and 78% of respondents in a new study believe that carriers should have access to gene therapy options.
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Gene Therapy for Rare Immune Disorder Gets FDA Review Date
October 25th 2023RP-L201 (marnetegragene autotemcel) is a one-time gene therapy that delivers a functional copy of the ITGB2 gene, which provides instructions for immune response to infections. The Prescription Drug User Fee Act (PDUFA) target action date is March 31, 2024.
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