Partnership Between Andelyn Biosciences and Grace Science Brings Hope to Patients with NGLY1 Deficiency

Partnership between Andelyn Biosciences and Grace Science to Enhance GS-100

Grace Science LLC has recently 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. This therapy is aimed at treating NGLY1 Deficiency, a serious and life-threatening disease with no approved treatment currently available. Patients with this disease experience debilitating symptoms throughout their lifetime.

Andelyn Biosciences is a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy, while Grace Science is focused on the development of novel therapies for rare diseases. The partnership between these two companies aims to maximize program efficiency, support ongoing clinical trials, and accelerate the availability of this critical therapy for patients suffering from NGLY1 Deficiency.

GS-100 is a recombinant AAV9 vector that encodes the human NGLY1 gene. In 2021, GS-100 received orphan drug designation from both the FDA and the European Medicine Agency, as well as Rare Pediatric Disease Designation from the FDA. Grace Science successfully dosed the first NGLY1 Deficiency patient with GS-100 earlier this year and is planning to dose the second patient in May 2024. As part of the partnership, Grace Science will benefit from Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial-readiness. This collaboration will help accelerate manufacturing timelines, bringing hope to patients in need of treatment.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed excitement about working with Grace Science and highlighted both companies’ commitment to quality and patient-centric focus. Matt Wilsey, CEO of Grace Science, also emphasized the importance of the partnership, noting that Andelyn’s expertise in AAV gene therapy manufacturing will ensure NGLY1 patients have access to safe and high-quality drug products.

The partnership between these two companies represents a significant step forward in bringing hope to patients with NGLY1 Deficiency by accelerating the availability of critical new therapies like GS-100.

In conclusion, Andelyn Biosciences Inc., a CDMO specializing in cell and gene therapy has recently partnered with Grace Science LLC to tech transfer and manufacture GS-100 for Phase I/II/III clinical trial material. GS-100 is an AAV suspension process gene therapy aimed at treating NGLY1 Deficiency which has no approved treatment currently available. The partnership aims to maximize program efficiency, support ongoing clinical trials and accelerate accessibility of critical therapies for patients suffering from this disease through expertise sharing between both companies while maintaining a commitment to quality standards.

Sophia Reynolds

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