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AI summary
Waskyra (etuvetidigene autotemcel) is a gene therapy for pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome who have a mutation in the WAS gene.
AI named Waskyra from January 2026 to August 2026.
Question: What are the latest affordable advancements in biopharma for treating rare diseases?
ChatGPT SearchAug 17, 2026
In December 2025, the FDA approved Waskyra, the first cell-based gene therapy for Wiskott-Aldrich syndrome.
What are the latest affordable advancements in biopharma for treating rare diseases?
AI named Waskyra most often alongside Center for Therapeutic Genetics, CASGEVY, and ENCELTO
ChatGPT SearchJun 22, 2026
Waskyra was approved in late 2025 as the first gene therapy for the rare immune disorder Wiskott–Aldrich syndrome.
AI cited fda.gov more than any other site in the answers that named Waskyra.
fda.gov/vaccines-blood-biologics/waskyra 25%Other sites 75%
Excerpts where Waskyra appeared in the AI's answer
In December 2025, the FDA approved Waskyra, the first cell-based gene therapy for Wiskott-Aldrich syndrome.
Waskyra was approved in late 2025 as the first gene therapy for the rare immune disorder Wiskott–Aldrich syndrome.
