AGC Biologics Celebrates U.S. and EU Approval of Fondazione Telethon’s Waskyra™ Treatment for Wiskott-Aldrich Syndrome

This further establishes the global leadership of AGC Biologics’ Milan Cell and Gene Center of Excellence in number of commercial products approved

MILAN–(BUSINESS WIRE)–#CGT–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 this vital, 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 on November 13, 2025, 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 organization to take on the commercialization 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.”

With its 30-year track record of regulatory approvals by the EMA and FDA, the AGC Biologics Milan site is a global leader with deep expertise in complex cell and gene therapy projects. The team has guided numerous products to commercial stages, manufactured hundreds of batches for clinical supply, and consistently met the highest global regulatory guidelines, quality performance metrics, and the unique complexities of technology transfers and manufacturing scale-up.

About Fondazione Telethon

Fondazione Telethon is an Italian non-profit biomedical organization committed to advancing research on rare and complex genetic diseases. For over 35 years, it has supported high-impact science aimed at developing innovative treatments and improving the lives of people affected by these conditions.

About AGC Biologics

AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to delivering the highest standard of service as we work side-by-side with our clients and partners, to provide friendly and expert services. We provide world-class development and manufacturing of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors, and genetically engineered cells. Our global network spans the U.S., Europe, and Asia, with locations in Seattle, Washington; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba and Yokohama, Japan. AGC Biologics is a part of AGC Inc.’s Life Science Business. The Life Science Business runs 10+ facilities focused on biopharmaceuticals, advanced therapies, small molecule active pharmaceutical ingredients, and agrochemicals. To learn more, visit www.agcbio.com.

Contacts

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