Grace Science and Andelyn Biosciences Partner for Late-Stage Manufacturing of Life-Saving Gene Therapy for NGLY1 Deficiency

Andelyn Biosciences Collaborates with Grace Science to Enhance GS-100

Grace Science LLC has partnered with Andelyn Biosciences Inc. 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.

Andelyn Biosciences Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), will utilize Grace Science’s expertise in late-stage manufacturing and commercial-readiness to accelerate their manufacturing timelines. 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.

In February of this year, Grace Science successfully dosed the first NGLY1 Deficiency patient with GS-100. The second patient is planned to be dosed in May 2024. 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. 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.

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.

This partnership is expected to help speed up clinical trials for GS-100 while ensuring safety standards are met. With more efficient processes in place

Sophia Reynolds

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