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Genezen and Charlotte & Gwenyth Gray Foundation Announcement

On April 28, 2026, Genezen—a contract development and manufacturing organization (CDMO) specializing in gene therapy—and The Charlotte & Gwenyth Gray Foundation to Cure Batten Disease announced a pivotal partnership aimed at bringing a new CLN6 gene therapy into clinical trials. This collaboration marks a significant step forward in the battle against CLN6 Batten disease, a rare and severe neurodegenerative disorder that currently has no approved therapies.

The Importance of This Partnership

This partnership is crucial, as Batten disease poses severe challenges to affected children and their families. With a focus on advancing a CLN6 gene therapy, the collaboration is dedicated to creating a treatment pathway for those impacted by this illness. The collaboration signals a more streamlined and efficient model for addressing the needs of ultra-rare disease programs, which historically face significant hurdles in gaining clinical traction.

Genezen has utilized its innovative viral vector manufacturing platform coupled with its regulatory expertise to support a seamless transition from proof of concept to Good Manufacturing Practice (GMP) compliance for clinical supply. The coordinated efforts of Genezen, the Gray Foundation, and their development partners have established a focused regulatory strategy. This includes direct engagement with the FDA to expedite the clinical process, which is vital for quickly moving forward with this promising therapy.

Voices of Hope

Dr. Susan D'Costa, Chief Technical and Commercial Officer at Genezen, expressed the company's commitment: "We are inspired by the Gray family's resilience and proud to move this program forward. 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."

In her statement, Kristen Gray, the Foundation's Founder and Director, noted, "We are thrilled to collaborate with Genezen in developing this vital therapy. Their proficiency, responsiveness, and collaborative spirit have been crucial in driving this initiative forward, fostering renewed hope for children suffering from CLN6 Batten disease."

A vital aspect of the program has been the engagement of a cross-functional team, including individuals like Tiffany Sepp, CEO of Vanguard Clinical, Inc., who is serving as the Development Team Leader for the Gray Foundation. The collaborative effort has highlighted the determination of the Gray family. After the tragic loss of their daughter Charlotte to this daunting disease, the family remains committed to finding treatment for their other daughter, Gwennie, and for all others affected by Batten disease.

Looking Ahead

Genezen is scheduled to present the regulatory and manufacturing strategies that underpin this program at the upcoming American Society for Gene and Cell Therapy annual meeting in May. This presentation aims to showcase not only the clinical progression but also the grounds for partnership success in addressing rare diseases through effective manufacturing and development practices.

This partnership illustrates Genezen's dedication to supporting parent-led foundations while providing efficient and scalable pathways for the treatment of rare patients. Their proven capabilities span the entire process from concept through commercialization, establishing a robust framework for delivering life-saving gene and cell therapies worldwide.

About Genezen

Genezen has over 12 years of experience as a leading gene therapy CDMO, equipped with advanced viral vector manufacturing facilities. Its mission is to partner with innovators to create transformative therapies, supporting life-saving advancements in medicine.

About The Charlotte & Gwenyth Gray Foundation

Founded to combat Batten disease, The Charlotte & Gwenyth Gray Foundation is committed to improving the lives of all impacted children. The foundation provides funding for research and financial support for families, highlighting the difficulties of living with neurodegenerative disorders. Their initiatives aim to ensure that families do not face the challenges of rare diseases alone.

For more information or to support this cause, please visit www.curebatten.org.