Andelyn Biosciences Collaborates with Grace Science to Enhance GS-100

Andelyn Biosciences Inc. is a cell and gene therapy Contract Development and Manufacturing Organization (CDMO) that has partnered with Grace Science LLC to tech transfer and manufacture GS-100, a suspension process AAV NGLY1 gene therapy for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency. This partnership aims to maximize program efficiency, support the ongoing clinical trial, and accelerate the critical therapy to patients suffering from NGLY1 Deficiency, a serious, life-threatening disease with no approved therapy that causes debilitating symptoms.

GS-100 is a recombinant AAV9 vector that encodes a full-length version of the human NGLY1 gene. It 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.

In the partnership, Grace Science will utilize Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial-readiness 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.

By Samantha Robertson

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