New Partnership Aims to Boost Efficiency and Deliver Life-Saving Gene Therapy for NGLY1 Deficiency

By Eleanor Harrison Mar25,2024
Andelyn Biosciences Collaborates with Grace Science to Further Develop GS-100

Grace Science LLC, a biopharmaceutical company focused on developing gene therapies for rare and life-threatening diseases, has announced a partnership with Andelyn Biosciences Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO). The collaboration aims to accelerate the development and manufacturing of GS-100, an AAV NGLY1 gene therapy intended for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.

NGLY1 Deficiency is a severe, life-threatening disease that currently has no approved therapy. Individuals with NGLY1 Deficiency endure debilitating symptoms throughout their lives. The partnership between Grace Science and Andelyn Biosciences seeks to improve efficiency, support ongoing clinical trials, and expedite the delivery of this crucial therapy to patients.

GS-100 is a recombinant AAV9 vector that contains a full-length version of the human NGLY1 gene. In 2021, GS-100 received orphan drug designation (ODD) from the FDA and the European Medicine Agency (EMA). Additionally, the FDA granted GS-100 Rare Pediatric Disease Designation in 2021. These designations open the possibility for Priority Review Voucher upon marketing approval and Fast-Track designation in 2023. Grace Science successfully administered the first dose of GS-100 to an NGLY1 Deficiency patient in February and plans to dose the second patient in May 2024.

Through this partnership, Grace Science will utilize Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness. This collaboration accelerates Grace’s manufacturing timelines, offering hope to individuals suffering from NGLY1 Deficiency.

Matt Wilsey, CEO and Co-Founder of Grace Science expressed excitement about this partnership with Andelyn Biosciences stating “Andelyn’s experience in AAV gene therapy manufacturing will ensure that our patients have access to a safe high quality drug product”. He emphasized on shared values of strong collaboration, customer centric focus, and commitment to quality which instills confidence in patients regarding success of GS – 100 program”. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences echoed Wilsey’s sentiments stating “We are thrilled about this opportunity to collaborate with Grace Science

By Eleanor Harrison

As a content writer at, I infuse flavor into words, crafting compelling stories that captivate and inform our audience. With a keen eye for detail and a passion for creativity, I strive to create content that not only engages but also inspires. Whether I'm concocting a savory blog post or whipping up a spicy product description, I pour my heart and soul into every piece I write. Join me on this flavorful journey as we explore the tantalizing world of content creation together.

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