Andelyn Biosciences Partners with Grace to Manufacture GS-100 for NGLY1 Gene Therapy

• Andelyn Biosciences has partnered with Grace Science LLC to manufacture GS-100, a gene therapy for NGLY1 Deficiency.

• GS-100 has been granted orphan drug designation by the FDA and EMA, and the first patient was dosed in February this year.

Andelyn Biosciences, a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has announced a partnership with Grace Science LLC. The collaboration aims to tech transfer and manufacture GS-100, a suspension process AAV NGLY1 gene therapy. The therapy is intended for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency, a serious, life-threatening disease with no approved therapy.

GS-100 is a recombinant AAV9 vector that encodes a full-length version of the human NGLY1 gene. In 2021, GS-100 was granted orphan drug designation (ODD) by the FDA and by the European Medicine Agency (EMA). It was also granted Rare Pediatric Disease Designation by the FDA in 2021, providing the potential for a Priority Review Voucher upon marketing approval and Fast-Track designation in 2023.

The first NGLY1 Deficiency patient was successfully dosed with GS-100 in February of this year. Grace Science is planning to dose the second patient in May 2024. As part of the partnership, Grace Science will leverage Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial-readiness.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed his enthusiasm about the partnership, stating, “We are privileged to work with a remarkable gene therapy company like Grace Science, whose tremendous dedication to rare diseases is an inspiration. Both Andelyn and Grace Science value strong collaboration and share a customer-centric focus and quality-first mindset. These synergies provide patients and their families confidence and hope for success of the GS-100 program.”