- Genezen has partnered with The Charlotte & Gwenyth Gray Foundation to manufacture a CLN6 gene therapy for a Phase 1/2 clinical trial.
- The collaboration supports the development of a treatment for CLN6 Batten disease, a rare neurodegenerative disorder with no approved therapies.
Genezen announced a manufacturing partnership with The Charlotte & Gwenyth Gray Foundation to Cure Batten Disease to advance a CLN6 gene therapy into a Phase 1/2 clinical trial. The collaboration focuses on developing a potential treatment for CLN6 Batten disease, a rare neurodegenerative condition that currently has no approved therapies.
The partnership combines Genezen’s viral vector manufacturing platform and regulatory expertise with the foundation’s development efforts. The companies stated that the program progressed from proof of concept to GMP clinical supply through coordinated efforts, including direct engagement with the U.S. Food and Drug Administration to support the regulatory pathway.
Genezen’s role as a CDMO includes providing contract manufacturing capabilities and regulatory support to accelerate the transition of the program into clinical development. The collaboration reflects a model aimed at advancing ultra-rare disease therapies through streamlined processes and cross-functional coordination.
“By combining regulatory insight with a flexible, platform-based manufacturing approach, our best-in-class team was able to accelerate this program into the clinic at a rapid pace.”
Susan D’Costa, Chief Technical and Commercial Officer at Genezen
The program involves collaboration with development partners, including leadership support from Vanguard Clinical, Inc. Genezen stated that the initiative aligns with its focus on supporting early-stage and foundation-led programs, enabling efficient development pathways from concept through clinical and commercial stages.
