This past weekend, our clinical team — Gai and Brandon — joined families for the FOXG1 Family Weekend Getaway at Morgan's Wonderland, not for a meeting, but to meet the kids and their families.

FOXG1 Research Foundation CFO and dad to Crosby reflects on what the $4.9M CIRM grant really means to the parent-led patient organization that started the journey with little hope for a future treatment, to now having a gene replacement therapy beginning first in-human clinical trial.

The FOXG1 Research Foundation has been awarded a $4,9M grant from the California Institute for Regenerative Medicine (CIRM) to advance FRF-001, our gene therapy, through its Phase 1/2 patient clinical trial.

"I can't even say this without getting a little teary." Watch Dr. Priscilla Chan talk about the FOXG1 Research Foundation’s gene therapy on Stanford Medicine's "The Future of Medicine."

Leaders from 14 countries across four continents gathered in Paris for the first-ever in-person FOXG1 Global Leadership Meeting — a historic moment eight years in the making, filled with gratitude, gene therapy updates, and a surprise $100,000 donation from Italy.

Nicole Johnson shared the FOXG1 story with new supporters in Paris, followed by a moving classical recital that included the live debut of "Who I Am" — a song written for children with FOXG1 by artist Isabella Coulstock.

Isabella Coulstock performed "Who I Am" as her voice filled the space, a video played — a montage of Josie's life, her happiness, her hard times, the big heart that takes her mommy's hand. FOXG1 families felt seen. Those newer to the cause felt the door open wide. Many were in tears. All of them reminded, in the span of a few minutes, exactly why this work matters — and who it is for.

Our team joined researchers and advocates at Rare Disease Day, where Dr. Soo Lee, Gai Ayalon, and CFO Ryan spoke on the science, development, and mission behind the FOXG1 gene therapy — capped by a poster session and dinner with the Lee lab.

FOXG1 families and Foundation leadership visited the Rockville CDMO site at Charles River Laboratories — where our gene therapy is being manufactured.

We saw firsthand the precision, safeguards, and care behind plasmid and AAV vector production, and the responsibility carried at every step.

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.

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.