Andelyn Biosciences and Grace Science, LLC Partner to Tech Transfer Phase I/II/III Manufacturing of a Suspension Process Adeno-Associated Virus (AAV) Gene Therapy for NGLY1 Deficiency

Media ReleaseNewsMarch 25, 2024

COLUMBUS, OH, March 25, 2024Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has 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 for the treatment of NGLY1 Deficiency. NGLY1 Deficiency is a serious, life-threatening disease with no approved therapy. Patients with this disease suffer from a lifetime of debilitating symptoms including developmental delay, cognitive impairment, (hypo) alacrimia, movement disorders, and other neurological symptoms. The partnership will seek to maximize program efficiency, support the ongoing clinical trial, and accelerate this critical therapy to patients.

GS-100 is a recombinant AAV9 vector that encodes a full-length version of the human NGLY1 gene. In 2021, GS-100 was granted orphan drug designation (ODD) by the FDA and by the European Medicine Agency (EMA). GS-100 was also granted Rare Pediatric Disease Designation by the FDA in 2021, providing the potential for a Priority Review Voucher upon marketing approval and Fast-Track designation in 2023. Grace Science successfully dosed the first NGLY1 Deficiency patient with GS-100 in February of this year and is planning to dose the second patient in May 2024.

As part of the partnership, Grace Science will leverage Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial-readiness. The partnership instills a quality mindset while also accelerating Grace’s manufacturing timelines, offering hope for patients suffering from NGLY1 Deficiency.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences said, “We are privileged to work with a remarkable gene therapy company like Grace Science, whose tremendous dedication to rare diseases is an inspiration. Both Andelyn and Grace Science value strong collaboration and share a customer-centric focus and quality-first mindset. These synergies provide patients and their families confidence and hope for success of the GS-100 program.”

Matt Wilsey, CEO and Co-Founder of Grace Science stated, “We are very excited about the partnership between Grace Science and Andelyn Biosciences. Andelyn’s deep expertise and experience in AAV gene therapy manufacturing will assure that our NGLY1 patients have access to safe, high-quality drug product for our clinical trial and beyond.”

Andelyn is advancing client discoveries and manufacturing life-altering cell and gene therapies for rare and prevalent diseases.  Founded on its extensive experience in AAV development and manufacturing, Andelyn offers its clients technology transfer capabilities, data-driven analytical-based process development and quality vector and plasmid manufacturing at its three Columbus, Ohio facilities.


About Andelyn Biosciences, Inc.
Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn’s deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing curative cell and gene therapies from concept through plasmid development and manufacturing, process development, and cGMP clinical and commercial manufacturing. Andelyn’s versatile capabilities include cGMP manufacturing capacity for both adherent and suspension processes up to a 2,000-liter capacity. An advanced digital model, quality system, full regulatory support and supply chain vertical integration help Andelyn accelerate the development and manufacturing of its clients’ innovative cell and gene therapies. For more information, visit

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About Grace Science
Grace Science, LLC is a biotechnology company founded in 2017 by Matt Wilsey and Dr. Carolyn Bertozzi to develop novel therapies based on the function of NGLY1, a key enzyme involved in proteostasis. The company’s deep knowledge of the NGLY1 pathway is key to its ability to identify new ways of treating NGLY1 Deficiency as well as more common diseases. For more information about Grace Science, LLC and NGLY1 Deficiency, please visit

For Grace Science media inquiries, please contact:
Brendan Beahm, Executive Director, R&D Operations, Grace Science, LLC,