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vs PBO & kept 57% vs 28% of pts in stage 2 Additionally, Sanofi is pausing its Teizeild application for recently diagnosed stage 3 T1D & is evaluating next steps with the EMA Company: Fondazione Telethon Product: Waskyra Active Ingredient: Etuvetidigene Autotemcel Disease: Wiskott-Aldrich Syndrome Date: Jan 09, 2026 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 patients CD34+ hematopoietic stem and progenitor cells, modified with a lentiviral vector carrying the WAS gene The ECs decision follows a CHMP opinion issued in November 2025 recommending marketing authorization, with the same therapy also approved by the U.S

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