June 13, 2024

BioPharmaceutical Innovations: Grace Science and Andelyn Biosciences Collaborate for Efficient Development and Manufacture of GS-100, Promising Therapy for NGLY1 Deficiency

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

Grace Science LLC, a biopharmaceutical company focused on rare genetic disorders, 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 this partnership, Andelyn Biosciences will bring its expertise in AAV processes and capabilities to late-stage manufacturing and commercial readiness. This collaboration will accelerate Grace’s manufacturing timelines, offering hope to individuals suffering from NGLY1 Deficiency.

Matt Wilsey, CEO and Co-Founder of Grace Science, expressed excitement about this partnership with Andelyn Biosciences: “We believe this collaboration will help us overcome some of the technical challenges we face in manufacturing our gene therapies.” He emphasized that Andelyn’s expertise will ensure that patients have access to safe, high-quality drug products for clinical trials and beyond.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed gratitude for this opportunity: “We are thrilled about this partnership with Grace Science as it aligns perfectly with our mission to improve patient outcomes through innovative solutions.” He emphasized that strong collaboration between both companies will help them deliver high-quality drug products quickly.

This partnership instills confidence and hope in patients suffering from NGLY1 Deficiency regarding the success of the GS-

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