- AGC Biologics has joined Orphan Therapeutics Accelerator’s Clinical Development Network as a manufacturing partner for ultra-rare disease therapies.
- The collaboration focuses on supporting cell and gene therapy development with integrated manufacturing and commercialisation capabilities.
AGC Biologics has joined the Clinical Development Network of Orphan Therapeutics Accelerator (OTXL) as a manufacturing partner to support the development and commercialisation of therapies for ultra-rare diseases.
The collaboration expands an existing relationship between the organisations and is intended to strengthen manufacturing support for cell and gene therapy programmes. OTXL said its Orphan ClinDevNet network brings together partners focused on reducing barriers associated with clinical development, manufacturing continuity, and commercial access for rare disease therapies.
According to the companies, AGC Biologics will contribute manufacturing expertise in ex vivo gene therapies, lentiviral vector production, and autologous CD34+ hematopoietic stem and progenitor cell platforms. The partnership builds on AGC Biologics’ previous work with Fondazione Telethon and the development of WASKYRA™, a gene therapy approved in December 2025.
“AGC’s expertise in ex vivo gene therapies and its ability to scale production through commercial supply gives us a manufacturing foundation that can support not only WASKYRA but also additional shelved programmes we take on.”
Craig Martin, CEO and Co-founder of Orphan Therapeutics Accelerator
AGC Biologics stated that its Milan facility has more than three decades of biologics manufacturing experience and has supported multiple viral vector and cell therapy approvals across the U.S. and Europe. The company said the collaboration aligns with its broader CDMO and contract manufacturing strategy focused on supporting commercially challenging therapies for small patient populations.
