The FDA Clears the FOXG1 Gene Replacement Therapy Clinical Trial to Begin!
FOR IMMEDIATE RELEASE
The U.S. Food and Drug Administration (FDA) Clears the Investigational New Drug (IND) Application for FRF-001, Clearing the Path to Initiate the FOXG1 Gene Replacement Therapy First-in-Human Clinical Trial
Port Washington, New York — January 24, 2026 — The FOXG1 Research Foundation (FRF) announced today 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 marks a historic milestone for rare disease drug development as the first instance of a parent-led rare disease nonprofit foundation to independently sponsor its own multi-site, international gene therapy clinical trial.
Without the prospect for pharmaceutical investment into therapeutic programs for FOXG1 syndrome, or the opportunity to secure government funding, the parent-led Foundation began pioneering a new model to efficiently drive rare disease drug development at a fraction of the traditional cost and timeframe.
“Today is the biggest milestone in FOXG1 Research Foundation history,” said Nicole Johnson, co-founder of the FOXG1 Research Foundation and mom to Josie (14). “For years, families like ours were told there was nothing we could do. We refused to accept that. This FDA clearance brings us into the next phase—moving our gene therapy through patient clinical trials—and brings real hope to families around the world.”
“This is parent-led drug development in action,” said Nasha Fitter, co-founder of the FOXG1 Research Foundation and mom to Amara (10). “We’ve shown there is another way to advance life-changing therapies for ultra-rare disorders.”
Fitter added, “As parents witnessing the absence of hope for our children’s rare disease as they struggle daily, and lives are lost, while seeing the advancements in genetic science, we knew there had to be another answer. Through innovation, rigor, efficiency, and the passion of parents, we’re proving that our nonprofit patient-led model allows us to accelerate timelines, and control critical decisions to protect the program from being put on the shelf as so often happens in the rare disease space.”
FOXG1 syndrome is an ultra-rare genetic neurodevelopmental disorder marked by profound developmental impairment, epilepsy, cognitive disabilities, and physical disabilities. FOXG1 is estimated to impact approximately 1 in 30,000 individuals worldwide, with emerging epidemiology suggesting prevalence may be higher.
FRF-001 is designed to address the underlying genetic cause of FOXG1 syndrome by delivering a functional copy of the FOXG1 gene using an AAV9 vector. The upcoming clinical trial will be conducted across multiple sites internationally and is being independently sponsored by the Foundation.
The preclinical gene therapy development is the result of countless hours at the FOXG1 Research Center (FRC) at the University at Buffalo, led by FOXG1 parents and neuroscientists Dr. Soo-Kyung Lee and Dr. Jae Lee. This is a unique story of parents who are also leading scientists—developing science from basic discovery to translational advancement at a dedicated academic center.
“Announcing the clearance of our IND for FRF-001 is the culmination of so much cross-functional work,” said Dr. Gai Ayalon, Chief Drug Development Officer of the FOXG1 Research Foundation. It takes the right team to pull this off in the non-traditional way we have, and we have that team. Being from industry for my entire career, doing this work now as a nonprofit is different and inspiring. We are writing a new playbook to bring treatments to patients with the greatest unmet need in medicine.”
The FOXG1 gene therapy program is privately funded through FRF’s “Yes, They Can!” campaign, a $22 million effort to advance FRF-001 through patient clinical trials and with a goal of regulatory approval. FRF has secured $14.5 million toward this goal and continues fundraising to close the remaining gap.
To share the news directly with the community, FOXG1 moms and Foundation co-founders Nasha Fitter and Nicole Johnson announced the milestone with a heartfelt video message, including next steps for parents and caregivers to stay informed and prepared as FRF advances FRF-001 through patient clinical trials.
Information pertaining to the clinical trial will be available on ClinicalTrials.gov
About the FOXG1 Research Foundation
The FOXG1 Research Foundation is a global, parent-led nonprofit organization founded in 2017 with the mission to improve the lives of every person and family impacted by FOXG1 syndrome. FRF advances research, builds community, and is pioneering a new blueprint for rare disease drug development—accelerating the path to life-changing therapies for children worldwide. Learn more at FOXG1Research.org.
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