Grace Science LLC Joins FDA START Program for GS-100 Therapy and Treats 2nd NGLY1 Patient

Grace Science, LLC recently announced a significant milestone: their investigational new drug (IND) for GS-100, an AAV9 gene therapy targeting NGLY1 Deficiency, has been accepted into the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program. This inclusion aims to expedite the drug development process for rare diseases through enhanced communication channels with the FDA, including initial and ad hoc meetings.

In May, Grace Science successfully treated the second patient with GS-100, following the first treatment earlier. Both patients, comprising the first cohort, are reportedly doing well without any adverse drug reactions. Plans are underway to increase the dosage for the third patient, who will form the second cohort, provided safety markers remain favorable.

Grace Science's CEO and Co-Founder, Matt Wilsey, expressed gratitude for being among the first companies accepted into the START Pilot Program. He emphasized the importance of regular dialogue with the FDA to streamline and hasten the development of GS-100 for NGLY1 Deficiency. Co-Founder Dr. Carolyn Bertozzi echoed this sentiment, highlighting GS-100's potential to revolutionize drug discovery and development, not just for NGLY1 Deficiency but for other rare and common diseases as well.

The GS-100 clinical trial, a Phase 1/2/3 study, aims to evaluate the long-term safety, tolerability, and efficacy of the gene replacement therapy. The Phase 1/2 segment will focus on dose-escalation and assess the safety and effectiveness of a single intracerebroventricular (ICV) infusion for patients aged 2-18 years across three dosage levels. The Phase 3 portion will measure the change from baseline in two primary outcomes at 52 weeks post-treatment: the NGLY1 Deficiency Primary Disease Activity Biomarker, GlcNAc-Asn (GNA, or N-acetylglucosamine) in cerebrospinal fluid, and the motor subdomain of the Bayley Scales of Infant and Toddler Development, Fourth Edition (BSID-4).

NGLY1 Deficiency is a critical, life-threatening condition for which no approved treatments currently exist. Affected patients exhibit severe symptoms from an early age, including global developmental delays, cognitive impairments, (hypo)alacrima, movement disorders, and various neurological issues.

Grace Science, LLC was founded in 2017 by Matt Wilsey and Dr. Carolyn Bertozzi. The biotechnology company is dedicated to developing innovative therapies based on the function of the NGLY1 enzyme, which plays a crucial role in proteostasis. Leveraging their extensive understanding of the NGLY1 pathway, Grace Science aims to discover new treatments not only for NGLY1 Deficiency but also for more prevalent diseases.