EMA Authorised: Waskyra (genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human Wiskott-Aldrich Syndrome (WAS) gene) — Fondazione Telethon ETS

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Summary

EMA authorised Waskyra, a gene therapy using autologous CD34+ cells modified with a lentiviral WAS gene, for Wiskott-Aldrich Syndrome.

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Diseases / indications

• Wiskott-aldrich Syndrome

Drugs mentioned

• Waskyra

Snippet

Status: Authorised. Therapeutic area: Wiskott-Aldrich Syndrome. MAH: Fondazione Telethon ETS. Decision date: 09/01/2026. Orphan medicine