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New FDA-Approved Gene Therapies

by Nikos Kokkorakis

A number of gene therapies that are being used for the first time have received approval from the US Food and Drug Administration (FDA). These treatments are for Dystrophic Epidermolysis Bullosa (DEB) (Vyjuvek /beremagene geperpavec-svdt, formerly known as B-VEC), for Duchenne muscular dystrophy (DMD) (SRP-9001 /delandistrogene moxeparvovec), and for hemophilia A (Roctavian /valoctocogene roxaparvovec). At the end of the previous year, it approved the first gene therapy for bladder cancer (Adstiladrin /nadofaragene firadenovec-vncg).

Concerning Dystrophic Epidermolysis Bullosa (DEB), Vyjuvek (beremagene geperpavec-svdt, formerly known as B-VEC) is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. Regarding Duchenne muscular dystrophy (DMD), SRP-9001 /delandistrogene moxeparvovec is a gene therapy to treat ambulant patients who are four to five years old with Duchenne muscular dystrophy (DMD) and have a confirmed mutation of the DMD gene dystrophin. As regards hemophilia A Roctavian /valoctocogene roxaparvovec is an investigational, one-time AAV-based gene therapy developed by BioMarin that delivers a working copy of the faulty gene into liver cells with instructions to produce the missing clotting factor. The gene therapy for bladder cancer is Adstiladrin /nadofaragene firadenovec-vncg, which is a non-replicating adenoviral vector-based gene therapy for the treatment of adult patients with high-risk, non-muscle-invasive bladder cancer (NMIBC).

 

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