The FOXG1 Research Foundation has named Brandon M. Henry, M.D., as Chief Medical Officer as FRF-001 advances toward a first-in-human clinical trial. Dr. Henry brings deep experience in AAV gene therapy development and will lead clinical strategy and oversight as we prepare to move into patient clinical trials.

As a parent-led organization serving as the independent sponsor of a multi-site, international gene therapy trial, FRF is pioneering a new rare disease model—advancing treatment with urgency while upholding the highest scientific and regulatory standards. Dr. Henry’s leadership strengthens our path forward as we work to bring a potential treatment to children and families impacted by FOXG1 syndrome.

The FOXG1 Research Foundation announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to FRF-001 for the treatment of FOXG1 syndrome — marking another major regulatory milestone as we advance toward our first-in-human Phase 1/2 clinical trial.

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.