3 Trial of GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency

2024-03-05

孤儿药快速通道基因疗法临床研究

MENLO PARK, Calif.--(BUSINESS WIRE)-- Grace Science, LLC announced today that the first patient was dosed in an open-label Phase 1/2/3 clinical trial of GS-100, an AAV9 gene replacement therapy for the treatment of NGLY1 Deficiency. Part 1/2 of the study will investigate dose-escalation and the safety and efficacy of a single intracerebroventricular (ICV) infusion of GS-100 in NGLY1 Deficiency patients aged 2-18 years. Part 3 of the study will assess the co-primary outcomes at 52 weeks following treatment at the selected dose: 1) change from baseline of the NGLY1 Deficiency Primary Disease Activity Biomarker, GlcNAc-Asn (GNA, or N-acetylglucosamine) in cerebrospinal fluid, and 2) change from baseline in the motor subdomain of the Bayley Scales of Infant and Toddler Development, Fourth Edition (BSID-4). Further details regarding the Phase 1/2/3 study can be found at . Matt Wilsey, CEO & Co-Founder of Grace Science, LLC, stated that "Treating the first patient is a tremendous milestone for our company and the NGLY1 Deficiency patients we strive to cure. We believe this drug will improve the lives of individuals living with this devastating disease. Time is precious as our community has lost sixteen individuals since January of 2020.” “This study is a true partnership between the scientific community and the NGLY1 patient community,” said Carolyn Bertozzi, Co-Founder of Grace Science, LLC. Dr. Bertozzi went on to say “The team has worked incredibly hard to reach the goal of treating the first NGLY1 Deficiency NGLY1 Deficiency patient with this potentially life-changing therapy. We are so thankful to the patients and their families for helping Grace Science turn this treatment into a reality.” About GS-100 GS-100 is a recombinant, single-stranded AAV9 vector that encodes a full-length version of the human NGLY1 gene. GS-100 was previously granted Orphan Drug Designation (ODD) by both the FDA and the European Medicine Agency (EMA) in 2021, Rare Pediatric Disease Designation by the FDA in 2021, and Fast Track Designation by the FDA in 2023. About NGLY1 Deficiency NGLY1 Deficiency is a serious, life-threatening disease with no approved therapy. Patients with this disease suffer from debilitating symptoms that present early in life, including global developmental delay, cognitive impairment, (hypo)alacrima, movement disorders, and other neurological symptoms. About Grace Science Grace Science, LLC is a biotechnology company founded in 2017 by Matt Wilsey and Dr. Carolyn Bertozzi to develop novel therapies based on the function of NGLY1, a key enzyme involved in proteostasis. The company’s deep knowledge of the NGLY1 pathway is key to its ability to identify new ways of treating NGLY1 Deficiency NGLY1 Deficiency as well as more common diseases. For more information about Grace Science, LLC and NGLY1 Deficiency, please visit gracescience.com.

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