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.