Parents Partner With Lab to Trial Gene Therapy for Batten

After losing their daughter Charlotte to CLN6 Batten disease, Kristen and her husband refused to give up on finding a cure for their other daughter, Gwennie, and other children battling this devastating condition. Now their foundation has partnered with Genezen, a gene therapy manufacturer, to bring an experimental treatment into Phase 1/2 clinical trials.

CLN6 Batten disease is a rare neurodegenerative disorder that robs children of their abilities over time. There are currently no approved treatments, leaving families with few options and little hope.

The Charlotte & Gwenyth Gray Foundation to Cure Batten Disease worked with Genezen's team to fast-track development from initial research to clinical-grade treatment supply. The collaboration included direct engagement with the FDA to speed up the approval process, using a streamlined approach designed specifically for ultra-rare diseases.

Susan D'Costa, Chief Technical and Commercial Officer at Genezen, praised the family's determination. The company's flexible manufacturing platform allowed them to accelerate the program at what she called "a rapid pace," cutting through typical delays that plague rare disease research.

Tiffany Sepp, who leads the development team for the Gray Foundation, highlighted the family's resilience. "The Grays are determined, even after the loss of their daughter Charlotte," she said. "They haven't given up on finding a treatment for their other daughter, Gwennie, and others impacted by Batten disease."

The Ripple Effect

This partnership represents more than one family's fight. It demonstrates a new model for how parent-led foundations can work with specialized manufacturers to bring treatments for ultra-rare diseases to reality faster than traditional pharmaceutical development allows.

The approach could open doors for other families facing similar diagnoses. Genezen plans to present their regulatory and manufacturing strategy at the American Society for Gene and Cell Therapy annual meeting in May, potentially creating a blueprint other organizations can follow.

Beyond funding research, the Gray Foundation provides financial support to families for education, therapies, and equipment needed for daily care. Their dual focus on finding a cure while supporting families today offers both immediate relief and long-term hope.

The clinical trial represents years of work compressed into an achievable timeline, proving that determination combined with the right partnerships can move mountains in medical research.