Goodwin congratulates Fondazione Telethon on receiving approval from the U.S. Food and Drug Administration (FDA) of its Biologics License Application for Waskyra™, an ex vivo gene therapy for patients with Wiskott-Aldrich syndrome, a rare and life-threatening immunodeficiency. This approval, the first ever granted by FDA for a cell and gene therapy product from a non-profit applicant, could serve as a model for making therapies available for rare diseases outside of the traditional industry pathway. Goodwin’s Life Sciences team is pleased to be working with Fondazione Telethon to bring this newly approved gene therapy product to patients in the U.S.
Fondazione Telethon is an Italian non-profit organization committed to advancing research on rare and complex genetic diseases. For over 35 years, it has supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions.
The Goodwin team working with Fondazione Telethon to launch the product in the U.S. is led by Julie Tibbets, Tim Atkins, Matt Wetzel, and Sue Abbott.
For more information on the approval, please read the press release.