AGC Biologics’ Milan Cell and Gene Center of Excellence will continue supporting the commercial development of Waskyra, following market authorization from the US FDA and the European Commission.
Waskyra is a gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening immune disorder affecting young patients worldwide.
A major milestone for rare disease gene therapy
The approvals further reinforce AGC Biologics Milan’s position as a global leader in commercial cell and gene therapy manufacturing. The site now supports one of the highest numbers of approved commercial cell and gene therapies globally.
About Wiskott-Aldrich syndrome
Wiskott-Aldrich syndrome is a rare genetic immune disorder.
- Affects ~1 in 250,000 live male births
- Symptoms begin in early childhood
- Includes severe infections, bleeding, and platelet dysfunction
Current options are limited to supportive care or stem cell transplantation from a suitable donor.
About Waskyra
Waskyra was developed by Fondazione Telethon, a long-term partner of AGC Biologics.
It is an ex vivo gene therapy using:
- Autologous CD34+ hematopoietic stem and progenitor cells
- Lentiviral vector–mediated gene correction
Waskyra received orphan drug designation in both the US and EU. On November 13, 2025, the CHMP issued a positive opinion, leading to EU marketing authorization.
AGC Biologics’ role in development and manufacturing
AGC Biologics supported Waskyra from preclinical to commercial stages. Its contributions included:
- Development and production of clinical-grade lentiviral vectors
- Manufacturing of patient-specific genetically engineered cells
- Regulatory support throughout development
This marks the third rare disease therapy where AGC continued manufacturing despite limited commercial viability due to ultra-small patient populations.
A long-standing partnership model
In 2023, Fondazione Telethon became the first non-profit organization to commercialize a gene therapy. AGC Biologics has supported Telethon’s ultra-rare disease pipeline for nearly two decades, spanning preclinical, clinical, and commercial phases.
Leadership perspectives
“For nearly 15 years, we’ve worked with Fondazione Telethon to produce the lentiviral vector and genetically modified cells behind this therapy,” said Luca Alberici, General Manager, AGC Biologics Milan. “We are fully committed to ensuring this life-changing therapy reaches every patient who needs it.”
“We are extremely proud of the FDA approval and the positive CHMP opinion,” said Celeste Scotti, Head of R&D at Fondazione Telethon. “AGC Biologics’ expertise was instrumental in bringing Waskyra to patients.”
Why Milan matters?
With a 30-year regulatory track record with both the EMA and FDA, the Milan site is a global reference point for complex therapies.
The team has:
- Guided multiple products to commercialization
- Manufactured hundreds of clinical batches
- Managed complex tech transfers and scale-ups
- Met the highest global quality and regulatory standards
About the organizations
Fondazione Telethon: An Italian non-profit biomedical organization advancing research in rare genetic diseases for over 35 years.
AGC Biologics: A global CDMO specializing in biologics, cell therapy, and gene therapy, working closely with partners to deliver complex medicines to patients worldwide.
