Fondazione Telethon's Waskyra™ Gene Therapy Gains CHMP Approval

Fondazione Telethon has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has given a positive opinion on Waskyra™, a gene therapy for Wiskott-Aldrich Syndrome (WAS). This condition is a severe genetic disorder affecting the immune system. Waskyra represents an ex vivo gene therapy, developed after decades of research at the San Raffaele Telethon Institute for Gene Therapy in Milan.

The organization highlights its achievement as a testament to the power of combining academic research with high industrial standards. This collaboration has led to a treatment option for a rare disease, highlighting a model where science meets patient care.

The therapy is expected to be available at IRCCS Ospedale San Raffaele, with a U.S. FDA review pending. It offers a potential option where other treatments might not be viable, emphasizing the need for accessible therapies.