• Tue. May 28th, 2024

Game-Changing Partnership Between Andelyn Biosciences and Grace Science Brings New Hope for NGLY1 Deficiency Patients

BySamantha Nguyen

Mar 25, 2024
Partnership between Andelyn Biosciences and Grace Science to Enhance GS-100

Andelyn Biosciences Inc. is a Contract Development and Manufacturing Organization (CDMO) that specializes in cell and gene therapy. The company has recently 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. 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.

The partnership between Andelyn Biosciences and Grace Science 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 Wilsey, CEO of Grace Science, 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. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, also expressed excitement about working with Grace Science and highlighted both companies’ commitment to quality and patient-centric focus.

By Samantha Nguyen

As a content writer at newsqwe.com, I am passionate about crafting engaging and informative articles that captivate our audience. With a background in journalism and a keen eye for detail, I strive to deliver content that is not only well-researched but also adds value to our readers' lives. From breaking news stories to in-depth features, I take pride in my ability to tell compelling stories that resonate with our diverse audience. When I'm not typing away at my keyboard, you can find me exploring new cafes, practicing yoga, or getting lost in a good book. I am thrilled to be a part of the newsqwe.com team and look forward to sharing my love for writing with all of our readers.

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