Genezen And The Charlotte & Gwenyth Gray Foundation To Cure Batten Disease Announce Manufacturing Partnership For CLN6 Gene Therapy Clinical Trial

Indianapolis, IN and Los Angeles, CA /PRNewswire/ - Genezen, a gene therapy contract development and manufacturing organization (CDMO), and The Charlotte & Gwenyth Gray Foundation to Cure Batten Disease today announced a manufacturing partnership to advance a CLN6 gene therapy into a Phase 1/2 clinical trial.

The collaboration aims to accelerate development of a potential treatment for CLN6 Batten disease, a rare neurodegenerative disorder with no approved therapies, and reflects a streamlined model for advancing ultra-rare disease programs.

Using its viral vector manufacturing platform and regulatory expertise, Genezen supported rapid progression from proof of concept to GMP clinical supply. Coordination across Genezen, the Gray Foundation, and development partners enabled a focused regulatory strategy, including direct engagement with the FDA to help expedite the path to clinic.

"We are inspired by the Gray family's resilience and proud to move this program forward," said Susan D'Costa, PhD, Chief Technical and Commercial Officer at Genezen. "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. Genezen is honored to partner with the Gray Foundation, and many other early stage/parent-founded biotechs to help cure rare diseases."

"We are thrilled to collaborate with Genezen in developing this vital therapy," stated Kristen Gray, Founder and Director of The Charlotte & Gwenyth Gray Foundation to Cure Batten Disease. "Their proficiency, responsiveness, and collaborative spirit have been crucial in driving this initiative forward, fostering renewed hope for children suffering from CLN6 Batten disease."

The program brought together a cross-functional team, including Tiffany Sepp, CEO of Vanguard Clinical, Inc., who serves as Development Team Leader for the Gray Foundation. "The Grays are determined, even after the loss of their daughter Charolotte to this extraordinarily challenging disease. They haven't given up on finding a treatment for their other daughter, Gwennie, and others impacted by Batten disease."

Genezen will present the regulatory and manufacturing strategy behind the program at the American Society for Gene and Cell Therapy annual meeting in May.

The partnership underscores Genezen's focus on supporting parent-led foundations and efficient, scalable pathways for N-of-1 and ultra-rare disease programs, backed by proven end to end capabilities from concept through commercialization.

About Genezen
Genezen is a best-in-class gene therapy CDMO with over 12 years of experience and state-of-the-art viral vector manufacturing facilities. From concept to commercial scale, Genezen partners with innovators to deliver life-saving gene and cell therapies worldwide. Learn more at genezen.com or follow Genezen on LinkedIn.

About The Charlotte & Gwenyth Gray Foundation to Cure Batten Disease
The Charlotte and Gwenyth Gray Foundation exists to improve and save the lives of all children impacted by Batten disease and other neurodegenerative disorders. Founded by two parents committed to finding a cure for Batten Disease to save the lives of their daughters, the Foundation recognizes firsthand the day-to-day challenges of living with a rare disease. In addition to funding research, the Foundation also provides financial support to families for education, patient therapies, and equipment critical to their well-being and rehabilitation. For more information and to support this cause, please visit www.curebatten.org