With the decision by the U.S. Food and Drug Administration and the European Commission to grant market authorization for Waskyra, a gene therapy to treat Wiskott-Aldrich syndrome, AGC Biologics’ Milan Cell and Gene Center of Excellence announced it will continue to support the commercial development of the life-changing therapy for young patients worldwide.
Wiskott-Aldrich syndrome is a rare immune disorder affecting approximately 1 in 250,000 live male births, leading to severe infections, increased bruising and bleeding, and other complications starting in early childhood due to problems with platelet production. Current treatment options include supportive therapies aimed at managing and preventing clinical manifestations, or a stem cell transplant from a familial donor.
Developed by Fondazione Telethon, a longtime client of AGC Biologics Milan, Waskyra is an ex vivo gene therapy using autologous CD34+ hematopoietic stem and progenitor cells that have been transduced with a lentiviral vector. Waskyra received orphan drug designation in both the EU/US and in November, the Committee for Medicinal Products for Human Use adopted a positive opinion of Waskyra that was sent to the European Commission to decide on EU marketing authorization.
To assist the effort to create a better treatment option for those afflicted by Wiskott-Aldrich syndrome, AGC Biologics developed and produced clinical grade lentiviral vectors carrying the relevant therapeutic gene and manufactured patient-specific genetically engineered cells from preclinical to commercial, assisting with regulatory filings at every step.
This adds to a growing list of cell and gene therapies manufactured by AGC Biologics approved for commercial use, and the third time AGC Biologics partnered with developers to continue manufacturing a rare disease drug considered not economically viable by traditional industry standards due to small population size and high manufacturing price per patient. In 2023, Fondazione Telethon became the first non-profit organisation to take on the commercialisation of a gene therapy, with AGC Biologics as their chosen manufacturer for lentiviral vectors and genetically engineered cells through the preclinical, clinical, and commercial development of their ultra-rare disease treatment pipeline for the last 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.”