With the FDA and European Commission granting market authorization for Waskyra, a groundbreaking gene therapy for Wiskott-Aldrich syndrome, AGC Biologics’ Milan Cell and Gene Center of Excellence is ramping up support to bring this life-changing treatment to young patients worldwide.
Wiskott-Aldrich syndrome, a rare immune disorder affecting roughly 1 in 250,000 male births, causes severe infections, bleeding, and other life-threatening complications from early childhood. Current options are limited to supportive therapies or stem cell transplants from familial donors.
Developed by Fondazione Telethon, Waskyra is an ex vivo gene therapy that uses patients’ own CD34+ hematopoietic stem and progenitor cells, genetically engineered with a lentiviral vector. The therapy earned orphan drug designation in the U.S. and EU, and on November 13, 2025, the European CHMP gave its positive opinion ahead of EU marketing authorization.
AGC Biologics played a central role in making Waskyra a reality, developing and producing clinical-grade lentiviral vectors and manufacturing patient-specific genetically engineered cells from preclinical stages through commercialization, while supporting regulatory filings at every step.
This marks the third time AGC Biologics has partnered on a rare disease therapy deemed commercially unviable by traditional industry standards due to small patient populations and high manufacturing costs. Fondazione Telethon became the first non-profit to commercialize a gene therapy in 2023, with AGC Biologics as its chosen manufacturing partner for nearly two decades.
“For nearly 15 years, we’ve worked with Fondazione Telethon to produce the lentiviral vector and the genetically modified cells that enable this therapy as part of our commitment to advancing innovative therapies for patients worldwide,” said Luca Alberici, General Manager, AGC Biologics Milan.
“Fondazione Telethon has an ethical responsibility to ensure this life-changing therapy reaches every patient who needs it and AGC is fully committed to deliver on this responsibility.”
“We are extremely proud of the FDA approval and the positive CHMP opinion for Waskyra™—milestones made possible also thanks to the high-quality services provided by AGC Biologics,” said Celeste Scotti, Head of Research and Development at Fondazione Telethon.
“Their expertise in producing lentiviral vectors and genetically modified cells has been instrumental in bringing this therapy to patients and in strengthening Fondazione Telethon's role as a leading player in the field of advanced therapies.”
