Grace Science and Andelyn Biosciences Team Up to Manufacture GS-100 for NGLY1 Deficiency: Collaboration Expedites Access to Life-Changing Therapy

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

Grace Science LLC and Andelyn Biosciences Inc., two leading players in the cell and gene therapy industry, have joined forces to manufacture GS-100. This collaboration aims to increase program efficiency, support ongoing clinical trials, and expedite the availability of this critical therapy to patients suffering from NGLY1 Deficiency, a debilitating condition with no approved treatment currently available.

NGLY1 Deficiency is a severe and life-threatening disease that affects patients with lifelong symptoms. The partnership between Andelyn Biosciences and Grace Science seeks to address this unmet medical need by leveraging Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness.

GS-100 is a recombinant AAV9 vector containing the full-length human NGLY1 gene. In 2021, it received orphan drug designation (ODD) from the FDA and the European Medicine Agency (EMA). The FDA also granted Rare Pediatric Disease Designation to GS-100 in 2021, potentially leading to a Priority Review Voucher upon approval for marketing and a Fast-Track designation in 2023. Grace Science administered the first dose of GS-100 to an NGLY1 Deficiency patient in February of this year and plans to treat a second patient in May 2024.

Through this partnership, Grace Science will leverage Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness to expedite its manufacturing timelines, providing hope for patients with NGLY1 Deficiency. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed his gratitude for the opportunity to collaborate with Grace Science. He highlighted their shared values of strong collaboration, customer-centric focus, and a quality-first mindset, which instills confidence and hope in the success of the GS-100 program.

Matt Wilsey, CEO and Co-Founder of Grace Science, emphasized his excitement about the partnership with Andelyn Biosciences. He noted that Andelyn’s expertise and experience in AAV gene therapy manufacturing would ensure that NGLY1 patients receive safe, high-quality drug products for clinical trials and beyond.

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