Shots:
- The CHMP has recommended Waskyra, 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 therapy will be available to pts at IRCCS Ospedale San Raffaele, where the clinical trial was conducted. The BLA for WAS remains under FDA review
Ref: PR Newswire| Image: Fondazione Telethon | Press Release
Related News:- The CHMP Recommends Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)
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