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gene therapy
Pharma
FDA signs off on 1st gene therapy for rare genetic disease
Fondazione Telethon's Waskyra is an ex vivo gene therapy that can address the root genetic cause of Wiskott-Aldrich syndrome.
Zoey Becker
Dec 10, 2025 12:09pm
ASH: CSL proves 5-year durability of Hemgenix
Dec 7, 2025 12:00pm
Novartis scores FDA approval for new version of SMA gene therapy
Nov 25, 2025 10:02am
FDA limits Elevidys gene therapy to ambulatory Duchenne patients
Nov 14, 2025 3:20pm
Catalent cuts 77 more jobs in Maryland
Nov 10, 2025 4:18pm
BioMarin looks to drop hemophilia gene therapy Roctavian
Oct 28, 2025 11:49am
