Andelyn Biosciences and Grace Science Partner for Expedited Manufacturing of GS-100 for NGLY1 Deficiency

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

In a recent development, Andelyn Biosciences Inc., a leading cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has partnered with Grace Science LLC to transfer technology and manufacture GS-100. This suspension process AAV NGLY1 gene therapy is intended for Phase I/II/III clinical trial material aimed at treating NGLY1 Deficiency, a debilitating condition without any approved therapy currently available.

NGLY1 Deficiency is a life-threatening disease that affects patients with life-long symptoms. The collaboration between Andelyn Biosciences and Grace Science aims 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 both 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 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.

Overall, this collaboration highlights the importance of collaboration between CDMOs like Andelyn Biosciences Inc., biotech companies like Grace Science LLC

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