CIRM Issues $60 Million in Grants Including Funding for FOXG1 Gene Therapy

Rare Daily Staff

California Institute of Regenerative Medicine, the state’s stem cell agency, is issuing $60 million in grants to support the development of experimental therapies, including a gene therapy for children with FOXG1 syndrome, a rare and severe neurological condition with no approved treatments.

The CIRM board approved nearly $21 million to support three early-stage clinical trials and more than $40 million for four preclinical programs. The investments are part of the agency’s long-running effort to move promising stem cell and gene therapies from the lab into human testing.

CIRM awarded about $4.9 million to support a phase 1/2 trial of FRF-001, an AAV9-based gene therapy designed to treat FOXG1 syndrome. The program is led by the FOXG1 Research Foundation, a patient-driven organization that has played a central role in advancing research on the condition.

FOXG1 syndrome is caused by mutations in a gene critical for brain development. Children with the disorder experience severe developmental delays, seizures, and limited speech and motor function.

The upcoming study will be an early-stage trial, primarily focused on safety while also looking for initial signs of benefit. Like many gene therapies, it aims to deliver a working copy of the FOXG1 gene to brain cells using a viral vector.

Two additional clinical-stage programs also received funding:

• An $8 million award to TR1X for a first-in-human trial of TRX319 in progressive multiple sclerosis, a form of the disease with limited treatment options.
• Nearly $8 million to Siren Biotechnology for SRN-101, an immuno-gene therapy targeting high-grade glioma, an aggressive brain cancer.

CIRM also committed more than $40 million to four preclinical programs, which are still in laboratory or animal testing but could eventually move into human trials. These include a functional cure for type 1 diabetes, a regenerative therapy to repair damage to hearts after a heart attack, a program to rebuild immune function using thymus cells derived from induced pluripotent stem cells, and a stem cell-engineered CAR-NKT cell therapy being explored for multiple sclerosis.