Grace Science LLC, a biotechnology company focused on developing gene therapies for rare diseases, has partnered with Andelyn Biosciences Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), to tech transfer and manufacture GS-100. GS-100 is a suspension process AAV NGLY1 gene therapy intended for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.
NGLY1 Deficiency is a severe, life-threatening disease that currently has no approved therapy. Individuals with NGLY1 Deficiency endure debilitating symptoms throughout their lives. The collaboration between Grace Science and Andelyn Biosciences aims to improve efficiency, support ongoing clinical trials, and expedite the delivery of this crucial therapy to patients.
GS-100 is a recombinant AAV9 vector that contains a full-length version of the human NGLY1 gene. In 2021, GS-100 received orphan drug designation (ODD) from the FDA and the European Medicine Agency (EMA). Additionally, the FDA granted GS-100 Rare Pediatric Disease Designation in 2021. These designations open the possibility for Priority Review Voucher upon marketing approval and Fast-Track designation in 2023. Grace Science successfully administered the first dose of GS-100 to an NGLY1 Deficiency patient in February and plans to dose the second patient in May 2024.
Through the partnership, Grace Science will utilize Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness. This collaboration accelerates Grace’s manufacturing timelines, offering hope to individuals suffering from NGLY1 Deficiency. Matt Wilsey, CEO and Co-Founder of Grace Science, conveyed excitement about the partnership with Andelyn Biosciences, highlighting the company’s expertise in AAV gene therapy manufacturing. He emphasized that Andelyn’s capabilities will ensure that NGLY1 patients have access to a safe, high-quality drug product for the clinical trial and beyond.
