• Sat. May 18th, 2024

Andelyn Biosciences and Grace Science Partner to Advance NGLY1 Gene Therapy for Patients with No Approved Treatment

BySamantha Jones

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

Andelyn Biosciences Inc. 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.

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 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.

Andelyn Biosciences Inc., based in Dallas Texas is a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy while Grace Science LLC is headquartered in Lexington Kentucky specializes in rare genetic diseases research & development

By Samantha Jones

As a dedicated content writer at newszxcv.com, I bring a passion for storytelling and a keen eye for detail to every piece I create. With a background in journalism and a love for crafting engaging narratives, I strive to deliver informative and captivating content that resonates with our readers. Whether I'm covering breaking news or delving into in-depth features, my goal is to inform, entertain, and inspire through the power of words. Join me on this journey as we explore the ever-evolving world of news together.

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