In a partnership aimed at accelerating the development of GS-100, a suspension process AAV NGLY1 gene therapy for the treatment of NGLY1 Deficiency, Andelyn Biosciences Inc. has partnered with Grace Science LLC. This collaboration will help maximize program efficiency, support ongoing clinical trials, and bring hope to patients suffering from this serious, life-threatening disease that has no approved therapy and causes debilitating symptoms.
GS-100 is a recombinant AAV9 vector that encodes a full-length version of the human NGLY1 gene and was granted orphan drug designation (ODD) by the FDA and EMA in 2021, along with Rare Pediatric Disease Designation by the FDA. Grace Science successfully dosed the first NGLY1 Deficiency patient with GS-100 in February of this year and plans to dose the second patient in May 2024.
The partnership between Andelyn Biosciences and Grace Science aims to bring safe, high-quality drug products to patients for their clinical trial and beyond. Through Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness, Grace Science will be able to accelerate their manufacturing timelines. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences emphasized the strong collaboration and customer-centric focus shared by both companies, providing confidence and hope for the success of the GS-100 program.
Matt Wilsey, CEO and Co-Founder of Grace Science expressed excitement about the partnership, stating that Andelyn’s expertise in AAV gene therapy manufacturing will ensure that NGLY1 patients have access to safe, high-quality drug products for their clinical trial and beyond. The partnership between Andelyn Biosciences and Grace Science aims to bring hope and relief to patients suffering from NGLY1 Deficiency through the development and manufacturing of GS-100.
