AGC Biologics joins rare disease therapy network as manufacturing partner

Orphan Therapeutics Accelerator (OTXL), a nonprofit biotechnology organization focused on advancing stalled rare disease therapies, announced that AGC Biologics has joined its Clinical Development Network as a manufacturing partner to support development and commercialization of ultra-rare disease treatments.

The collaboration expands an existing relationship between the organizations and is intended to strengthen manufacturing support for cell and gene therapy programs facing commercialization and scale-up challenges tied to small patient populations and high production costs.

OTXL said its Orphan ClinDevNet initiative brings together specialized partners aimed at reducing development costs, supporting late-stage clinical programs, and improving manufacturing continuity for ultra-rare therapies.

“Despite major advances in cell and gene therapy, the commercial sustainability of treatments for ultra-rare diseases remains a critical hurdle, preventing many proven therapies from reaching patients,” said Luca Alberici, vice president of the Cell and Gene Technologies Division at AGC Biologics, in a statement. “At AGC Biologics, we have built the specific expertise and infrastructure to sustainably manufacture these products.”

The collaboration builds on AGC Biologics’ existing work with Fondazione Telethon on WASKYRA, a gene therapy approved by the FDA in December 2025. According to the companies, Fondazione Telethon became the first nonprofit organization to receive FDA approval for a gene therapy, with AGC Biologics serving as manufacturer and OTXL subsidiary Orphan Therapies supporting U.S. commercialization.

The announcement follows AGC Biologics’ broader expansion efforts in Japan. In April 2026, the company announced plans for a $350 million manufacturing facility in Yokohama designed to expand mammalian, cell therapy, and mRNA production capacity in the Asia-Pacific region. The GMP-ready site, expected to begin operations in 2027, will include one of Japan’s largest single-use mammalian bioreactor systems and support both clinical and commercial manufacturing.

OTXL said AGC Biologics’ global manufacturing footprint will help support geographically dispersed patient populations affected by ultra-rare diseases while enabling broader access to advanced therapies.