The FOXG1 Research Foundation announced the FDA has granted Orphan Drug and Rare Pediatric Disease designations to FRF-001, its gene therapy for FOXG1 syndrome. FRF-001 marks a historic first: a parent-led foundation sponsoring its own international, multi-site clinical trial.

Port Washington, NY September 29, 2025 --(PR.com)-- The FOXG1 Research Foundation Announces FDA Award of Orphan Drug Designation for FRF-001 Gene Therapy, Following Rare Pediatric Disease Designation

www.foxg1research.org

FRF-001 — named for the FOXG1 Research Foundation itself — reflects a rare and historic milestone, as a parent-led foundation sponsors its own global clinical trial. This designation follows FDA’s earlier award of Rare Pediatric Disease designation to FRF-001.

The FOXG1 Research Foundation (FRF) today announced a major milestone in its journey toward a clinical treatment for FOXG1 syndrome. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to FRF-001, the Foundation’s lead gene therapy candidate for the treatment of FOXG1 syndrome. This follows the FDA’s earlier award of Rare Pediatric Disease (RPD) designation to the same investigational therapy.

FRF-001 is being developed under the Foundation’s stewardship, upon IND approval, the FRF will be sponsoring its multi-site, international clinical trial independently — a historic first for a parent-led rare disease foundation.

“This designation signifies a favorable path forward and demonstrates the FDA’s recognition of the potential of FRF-001,” said Dr. Gai Ayalon, Chief Drug Development Officer at FRF. “We look forward to continuing our close dialog with FDA as we advance toward first-in-human trials.”

About FOXG1 syndrome and FRF’s mission
FOXG1 syndrome is a rare, devastating neurodevelopmental disorder caused by mutations in the FOXG1 gene. Affected children experience a spectrum of severe neurological symptoms, including developmental delays, movement disorders, seizures, and intellectual disability. Currently, there is no approved disease-modifying therapy.

The FOXG1 Research Foundation www.foxg1research.org was founded in 2017 by parents to accelerate the development of effective treatments and to bring hope to families impacted by FOXG1 syndrome. Through partnerships, scientific collaboration, and strategic investment, FRF aims to become the sponsor of first-in-human studies for FRF-001 and ultimately deliver a safe and transformative therapy for patients.

Forward-looking statements
This press release contains forward-looking statements, including statements about the potential benefits and timing of regulatory designations, clinical trials, and future approvals. These statements are subject to risks and uncertainties, including the possibility that FRF-001 may not successfully complete clinical development or receive regulatory approval, that the projected benefits of ODD or RPD designation may not materialize, or that legislative or regulatory changes could affect the incentives described. Actual results may differ materially from those expressed or implied herein.

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Contact Information:
FOXG1 Research Foundation
Nicole Johnson
Contact via Email
www.foxg1research.org

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