Game-Changing Partnership Set to Revolutionize Treatment for NGLY1 Deficiency with AAV Gene Therapy Manufacturing

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

NGLY1 Deficiency is a life-threatening disease with no approved therapy. Individuals suffering from this condition endure debilitating symptoms throughout their lives. Grace Science LLC, a company focused on gene therapy, has partnered with Andelyn Biosciences Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO) to tech transfer and manufacture GS-100, an AAV NGLY1 gene therapy intended for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.

The collaboration between these two companies 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.

Through this partnership, Grace Science will utilize Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness. This collaboration will accelerate Grace’s manufacturing timelines, offering hope to individuals suffering from NGLY1 Deficiency. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed gratitude for the opportunity to collaborate with Grace Science and emphasized the shared values of strong collaboration, customer-centric focus, and commitment to quality. This partnership instills confidence and hope in patients and their families regarding the success of the GS-100 program. 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.

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