Maria (Masha) Babak, Head of The Babak Lab and Assistant Professor at City University of Hong Kong, shared The Babak Lab’s post on LinkedIn, adding:
“Great example of non-profit science making real clinical impact.”
Quoting The Babak Lab’s post:
“Clinical Mondays: FDA Approves First Gene Therapy for Wiskott-Aldrich Syndrome
The U.S. Food and Drug Administration (FDA) has approved Waskyra (etuvetidigene autotemcel) – the first cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening genetic immunodeficiency.
Key Focus
Waskyra is indicated for pediatric patients aged six months and older and adults with a mutation in the WAS gene for whom hematopoietic stem cell transplantation (HSCT) is appropriate but no suitable matched related donor is available. The therapy uses the patient’s own genetically modified hematopoietic stem cells to restore functional WAS protein expression and address the root cause of the disease.
Key Insights
- Waskyra represents the first FDA-approved gene therapy for Wiskott-Aldrich Syndrome – and the first cell and gene therapy product approved from a non-profit sponsor (Fondazione Telethon ETS).
- In clinical studies and expanded access programs, treatment resulted in substantial and sustained clinical benefits, including a 93% reduction in severe infections and 60% reduction in moderate to severe bleeding following treatment.
Conclusion
This approval is a major win for patients with Wiskott-Aldrich syndrome and a testament to the outstanding work of the Telethon scientists. Hopefully, it will make a real difference in patients’ lives.”
More posts featuring Maria Babak.