FOXG1 Research Foundation Enters Collaboration with CRL to Advance Gene Therapy

Rare Daily Staff

The FOXG1 Research Foundation has entered into a collaboration with Charles River Laboratories International for the foundation’s experimental gene therapy to treat the rare, neurodevelopmental condition FOXG1 syndrome.

Under the collaboration, Charles River will provide the foundation with access to cell and gene therapy expertise and generate materials for the organization’s phase 1/2 adeno-associated viral vector-based gene therapy clinical trials.

FOXG1 syndrome is a severe rare neurological genetic disorder that greatly impacts early brain development and typically causes epilepsy and a host of medical complexities and disabilities. There are approximately 1,000 patients diagnosed with FOXG1 syndrome worldwide, with the diagnosis rate climbing steadily year-over-year and no approved treatments. Most children with FOXG1 syndrome cannot walk or talk or take care of their basic needs.

The experimental therapy in development by the foundation is an AAV vector gene replacement therapy that is designed to insert a healthy copy of the FOXG1 gene into cells in the body of individuals living with FOXG1 syndrome to replace the loss of FOXG1 protein caused by the mutations. The foundation is simultaneously pursuing other therapeutic approaches including an antisense oligonucleotide therapy, an RNAi therapy, a tRNA therapy, and a CRISPRa therapy.

The collaboration with Charles River is an example of how organizations for ultra-rare diseases are increasingly moving beyond funding basic research to take a hands-on approach to therapeutic development to de-risk development to a point where a biopharmaceutical company would be willing to take over development or commercialization.

“Given the limited investment for rare disease groups like ours, our foundation has created a model that allows us and other patient advocacy groups to operate like a virtual biotech company and independently and efficiently drive drug development,” said Nasha Fitter, FOXG1 Parent, Co-founder and CEO of the FOXG1 Research Foundation

Photo: Nasha Fitter, co-founder and CEO of the FOXG1 Research Foundation