The FDA Clears the FOXG1 Gene Replacement Therapy Clinical Trial to Begin!  

The FOXG1 Research Foundation announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to begin first-in-human clinical trials for FRF-001, the first FOXG1 AAV9 gene replacement therapy. This historic milestone marks the first instance of a parent-led rare disease nonprofit foundation independently sponsoring its own multi-site, international gene therapy clinical trial.