Shots:
- The EC has approved Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS)
- Waskyra is a single-administration therapy that uses a patient’s CD34+ hematopoietic stem and progenitor cells, modified with a lentiviral vector carrying the WAS gene
- The EC’s decision follows a CHMP opinion issued in November 2025 recommending marketing authorization, with the same therapy also approved by the U.S. FDA in December 2025; underscoring the effectiveness and global uniqueness of its research and development model
Ref: Fondazione Telethon | Image: Fondazione Telethon | Press Release
Related News:- Fondazione Telethon’s Waskyra Receives the CHMP’s Positive Opinion for WAS
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