Grace Science, LLC announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GS-100, an investigational AAV9 gene replacement therapy for the treatment of NGLY1 deficiency.

The FDA grants orphan drug designation to investigational medicines and biologics intended for the safe and effective treatment, diagnosis, or prevention of rare disease or conditions affecting fewer than 200,000 people in the United States.

Orphan drug designation provides certain benefits and incentives to Grace Science for developing a gene therapy for NGLY1 deficiency, including seven years of U.S. marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain clinical research costs, eligibility for orphan drug grants, and a waiver of the Prescription Drug User Fee Act (PDUFA) filing fees.

NGLY1 deficiency is an ultra-rare genetic disease caused by loss of function mutations in the NGLY1 gene. The disease primarily affects children and manifests as a complex neurological syndrome that includes global developmental delay, cognitive impairment, movement disorders, abnormal tear production (alacrima), as well as other neurological symptoms. There is currently no approved therapy for NGLY1 deficiency.

Matt Wilsey, founder and CEO of Grace Science stated: “We are pleased the FDA has granted orphan drug designation to our gene therapy program for NGLY1 deficiency. This action highlights the significant need that exists for the development of safe and effective treatments for children suffering from this condition, including my own daughter, Grace. Our recent interactions with the FDA have been highly productive and we are making rapid progress on our plans to file an investigational new drug application (IND) in the third quarter of Q3 2022, which would allow us to initiate our first clinical trial by the end of next year.”

Grace Science recently closed its Series A financing, which will fund the advancement of GS-100 into a first-in-human clinical trial for NGLY1 deficiency. The viral vector development and clinical manufacturing services for GS-100 will be provided by Thermo Fisher Scientific at its site in Alachua, FL.