Breakthrough in NGLY1 Deficiency Treatment: Grace Science and Andelyn Biosciences Partner for Late-Stage Manufacturing and Commercial Readiness

Andelyn Biosciences Collaborates with Grace Science to Further Develop GS-100

Grace Science LLC, a biopharmaceutical company focused on the development of gene therapies for rare diseases, has entered into a partnership 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 Andelyn Biosciences and Grace Science 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 this partnership, Andelyn Biosciences will provide its expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness to help accelerate Grace’s manufacturing timelines. This collaboration provides hope for individuals suffering from NGLY1 Deficiency who may now have access to a safe, high-quality drug product for clinical trial use.

Matt Wilsey, CEO and Co-Founder of Grace Science expressed excitement about this partnership with Andelyn Biosciences as it highlights their expertise in AAV gene therapy manufacturing. He emphasized that this collaboration will ensure that NGLY1 patients have access to a safe, high-quality drug product for both clinical trials and beyond.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences said he was thrilled about collaborating with Grace Science on this project as they share similar values such as strong collaboration, customer focus

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