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

Grace Science LLC has partnered with Andelyn Biosciences Inc., a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy, 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.

Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial-readiness will benefit Grace Science as part of this collaboration. 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 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.

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. The partnership between these two companies is expected to continue beyond these initial trials as they work towards bringing this critical therapy to more patients who are suffering from this debilitating disease.

By Samantha Johnson

As a content writer at, I craft engaging and informative articles that aim to captivate readers and provide them with valuable insights. With a background in journalism and a passion for storytelling, I thoroughly enjoy delving into diverse topics, conducting research, and producing compelling content that resonates with our audience. From breaking news pieces to in-depth features, I strive to deliver content that is both accurate and engaging, constantly seeking to bring fresh perspectives to our readers. Collaborating with a talented team of editors and journalists, I am committed to maintaining the high standards of journalism upheld by our publication.

Leave a Reply