Partnership Between Andelyn Biosciences and Grace Science Brings Hope to Patients Suffering from NGLY1 Deficiency through Development of GS-100 Gene Therapy

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

Grace Science LLC and Andelyn Biosciences Inc., a leading cell and gene therapy Contract Development and Manufacturing Organization (CDMO), have announced a strategic partnership to tech transfer and manufacture GS-100, an AAV NGLY1 gene therapy intended for clinical trial material. The collaboration aims to improve efficiency, support ongoing clinical trials, and expedite the delivery of this crucial therapy to patients suffering from NGLY1 Deficiency.

NGLY1 Deficiency is a severe, life-threatening disease with no approved treatment. Individuals with NGLY1 Deficiency experience debilitating symptoms throughout their lives. With the partnership between Andelyn Biosciences and Grace Science, the hope for those affected by NGLY1 Deficiency is growing.

GS-100 is a recombinant AAV9 vector containing a full-length version of the human NGLY1 gene. In 2021, it received orphan drug designation (ODD) from both the FDA and EMA. Additionally, the FDA granted GS-100 Rare Pediatric Disease Designation in 2021, opening 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 2024, with plans to dose the second patient in May 2024.

The partnership between Andelyn Biosciences and Grace Science will allow Grace Science to 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 who are eagerly awaiting access to safe, high-quality drug products for clinical trials and beyond.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed his gratitude for the opportunity to collaborate with Grace Science while emphasizing the shared values of strong collaboration, customer-centric focus, and commitment to quality.

Matt Wilsey, CEO and Co-Founder of Grace Science expressed excitement about this partnership with Andelyn Biosciences highlighting their expertise in AAV gene therapy manufacturing that will ensure that patients affected by NGLY1 Deficiency have access to safe high quality drug product for clinical trials.

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