AGC Biologics’ Milan Cell and Gene Centre of Excellence has announced continued support for the commercial development of Waskyra, a gene therapy for Wiskott-Aldrich syndrome, following market authorisation decisions by the US Food and Drug Administration (FDA) and the European Commission.

The approvals further reinforce AGC Biologics’ global leadership in cell and gene therapy manufacturing, with the Milan site continuing to expand its portfolio of commercially approved advanced therapy products.

Wiskott-Aldrich syndrome is a rare, inherited immune disorder affecting approximately one in 250,000 live male births. The condition leads to severe infections, abnormal bleeding, and other complications beginning in early childhood due to impaired platelet production. Current treatment options are limited to supportive care or stem cell transplantation from a suitable familial donor.

Luca Alberici, General Manager of AGC Biologics Milan, said, “For nearly 15 years, we have worked closely with Fondazione Telethon to produce the lentiviral vectors and genetically modified cells that enable this therapy. Our shared commitment is to ensure this life-changing treatment reaches every patient who needs it.”

Celeste Scotti, Head of Research and Development at Fondazione Telethon, added, “AGC Biologics’ expertise in advanced therapy manufacturing was instrumental in achieving regulatory milestones and delivering the therapy to patients.”

Developed by Fondazione Telethon, a long-term partner of AGC Biologics, Waskyra is an ex vivo gene therapy that uses a patient’s own CD34 plus hematopoietic stem and progenitor cells, genetically modified using a lentiviral vector. The therapy received orphan drug designation in both the US and the EU. On November 13, 2025, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, paving the way for European Commission marketing authorization.

AGC Biologics has supported the programme from early development through commercialisation by producing clinical-grade lentiviral vectors and manufacturing patient-specific genetically engineered cells. The company also assisted with regulatory submissions throughout the development lifecycle.

The Waskyra approval adds to a growing list of cell and gene therapies manufactured by AGC Biologics that have achieved commercial authorisation. It also marks the third time the company has partnered on a rare disease therapy considered economically challenging due to small patient populations and high manufacturing costs. In 2023, Fondazione Telethon became the first non-profit organisation to commercialise a gene therapy, selecting AGC Biologics as its manufacturing partner across its ultra-rare disease pipeline.