FRF-001 is an investigational AAV9 gene replacement therapy developed by the FOXG1 Research Foundation for FOXG1 syndrome. FDA cleared the IND in January 2026 to begin first-in-human Phase 1/2 testing, and later granted Fast Track designation. The program is being independently sponsored by a parent-led rare disease nonprofit.
The FDA has approved nivolumab with chemotherapy for pediatric and adult Hodgkin lymphoma patients aged 12+, based on clinical trial data showing improved survival. Separately, the agency cleared investigational new drug applications for FG001 for brain cancer surgery visualization and FRF-001 gene therapy for FOXG1 syndrome, allowing both to proceed to clinical trials.
The U.S. Food and Drug Administration has approved an Investigational New Drug application for FRF-001, a viral gene therapy for FOXG1 syndrome developed at the University at Buffalo. The first-in-human trial will be independently sponsored by the FOXG1 Research Foundation.
