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Game-Changing Partnership Aims to Improve Efficiency and Deliver Crucial Therapy for NGLY1 Deficiency

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

Game-Changing Partnership Aims to Improve Efficiency and Deliver Crucial Therapy for NGLY1 Deficiency

Grace Science LLC, a pioneering biotechnology company, has partnered with Andelyn Biosciences Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), to tech transfer and manufacture GS-100. GS-100 is a suspension process AAV NGLY1 gene therapy intended for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.

NGLY1 Deficiency is a severe and life-threatening disease that affects individuals with an inherited genetic mutation in the N-glycosyltransferase 1 (NGLY1) gene. The condition causes debilitating symptoms throughout life, including muscle weakness, liver failure, and other organ dysfunctions. With no approved therapy currently available, patients with NGLY1 Deficiency face an uncertain future without intervention.

The collaboration between Andelyn Biosciences and Grace Science aims to improve efficiency in manufacturing processes, support ongoing clinical trials, and expedite the delivery of this crucial therapy to patients. By leveraging their combined expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness, Grace Science can accelerate its timelines for producing GS-100. This development offers hope to individuals suffering from NGLY1 Deficiency who are eagerly awaiting access to a safe, high-quality drug product for the clinical trial stage and beyond.

In February 2024, Grace Science successfully administered the first dose of GS-100 to an NGLY1 Deficiency patient as part of ongoing clinical trials. The second dose will be given in May 2024. GS-100 received orphan drug designation (ODD) from both the FDA and EMA in 2021. Additionally, the FDA granted GS-100 Rare Pediatric Disease Designation in 2021. These designations open up possibilities such as Priority Review Voucher upon marketing approval and Fast-Track designation in 2023, allowing for expedited development and review processes for new therapies treating rare diseases like NGLY

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