• Sun. May 26th, 2024

Collaboration between Grace Science and Andelyn Biosciences Boosts Efficiency, Expedites Manufacturing of GS-100 for NGLY1 Deficiency Patients

BySamantha Nguyen

Mar 25, 2024
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 collaboration aims to maximize program efficiency, support the ongoing clinical trial, and accelerate the critical therapy to patients suffering from this serious, life-threatening disease with no approved treatment that causes debilitating symptoms.

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. In February of this year, Grace Science successfully dosed the first NGLY1 Deficiency patient with GS-100 and plans to dose the second patient in May 2024.

In this partnership, Grace Science will utilize Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial-readiness to accelerate their manufacturing timelines. 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.

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. 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.

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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