- AGC Biologics’ Milan Cell and Gene Center of Excellence will continue supporting the commercial development of Waskyra™, an approved gene therapy for Wiskott-Aldrich syndrome.
- The company manufactured lentiviral vectors and patient-specific genetically engineered cells from preclinical stages through to commercial supply.
AGC Biologics has confirmed it will continue to support the commercial development of Waskyra™, following market authorisation decisions by the U.S. Food and Drug Administration and the European Commission. The gene therapy is intended to treat Wiskott-Aldrich syndrome, a rare inherited immune disorder affecting approximately one in 250,000 live male births.
Waskyra was developed by Fondazione Telethon, a long-standing partner of AGC Biologics’ Milan site. The therapy is an ex vivo gene therapy using autologous CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector. In November 2025, the Committee for Medicinal Products for Human Use adopted a positive opinion, which was subsequently sent to the European Commission for a decision on EU marketing authorisation.
As part of the programme, AGC Biologics developed and produced clinical-grade lentiviral vectors carrying the therapeutic gene and manufactured patient-specific genetically engineered cells. The company supported the programme from preclinical development through clinical stages to commercial manufacturing, alongside regulatory submissions at each phase. This work was carried out at the company’s Milan Cell and Gene Center of Excellence as part of its CDMO and contract manufacturing services for advanced therapies.
The approval adds to a growing list of cell and gene therapies manufactured by AGC Biologics that have reached commercial use. It also marks the third occasion on which the company has partnered on a rare disease therapy that is considered challenging to commercialise due to small patient populations and high per-patient manufacturing costs.
“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.”
Luca Alberici, General Manager of AGC Biologics Milan
