Grace Science and Andelyn Biosciences Join Forces to Develop GS-100, Promising Gene Therapy for NGLY1 Deficiency

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

Grace Science LLC and Andelyn Biosciences Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), have joined forces to transfer technology and manufacture GS-100. GS-100 is a suspension process AAV NGLY1 gene therapy intended for Phase I/II/III clinical trial material aimed at treating NGLY1 Deficiency, a serious and life-threatening disease without any approved therapy currently available.

NGLY1 Deficiency is a debilitating condition that afflicts patients with life-long symptoms. The collaboration between Andelyn Biosciences and Grace Science seeks to increase program efficiency, support ongoing clinical trials, and expedite the availability of this crucial therapy to patients in need.

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

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed 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.

In conclusion, this partnership between two leading organizations in cell and gene therapy manufacturing will bring new hope to patients suffering from NGLY1 Deficiency by accelerating the development of safe

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