Isabella Coulstock had never performed Who I Am live before that night in Paris.

When she finally did, the room went still.

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.

Rare Disease Day at the University at Buffalo reminded us of something we never want to take for granted: what becomes possible when science, urgency, and mission are truly aligned.

From Dr. Soo Lee's presentation on the foundational science behind our AAV9 gene therapy, to Gai Ayalon's candid look at the complex journey from academic discovery to clinical-stage therapeutic, to the inspiring poster session showcasing the depth of research underway in the Lee lab — the day was a powerful testament to what a community united around a common goal can achieve.

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.

We are deeply grateful for this partnership and the commitment to bringing treatment to children with FOXG1 syndrome.

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.

WILMINGTON, Mass.--(BUSINESS WIRE)--Jul. 30, 2024-- Charles River Laboratories International, Inc. (NYSE: CRL) announced today a collaboration with the FOXG1 Research Foundation (FRF) highlighting the patient advocacy group’s model to independently drive drug development through the clinical phase. The parent-led global organization driving the research to cure FOXG1 syndrome and related neurological disorders will collaborate with Charles River in a comprehensive gene therapy contract development and manufacturing organization (CDMO) agreement.

“Charles River is proud to work with the FOXG1 Research Foundation to advance its gene-therapy through clinical trials,” said Kerstin Dolph, Corporate Senior Vice President, Global Manufacturing, Charles River. “The FOXG1 patient population has an incredible unmet need, and we are looking forward to lending our expertise to FRF as they continue to trailblaze a path toward providing rare disease treatments.

2023 marked six years of the FOXG1 Research Foundation. We are a parent-driven, global foundation with the mission to help improve the lives of every person affected by FOXG1 syndrome worldwide. We are known in the industry as innovators as we  focus on three equally critical areas: FOXG1 science, FOXG1 patient data, and FOXG1 patients and community. From advancing our gene therapy program to building our own bioinformatics platform, to helping guide parents through the rare disease medical caregiver journey, this year has been a year of upward progress in all three areas. Here are the FOXG1 Research Foundation highlights from 2023 and goals for 2024.