Andelyn Biosciences and Grace Science Partner on Gene Therapy for Rare Disease

26 March 2024 | Tuesday | News

The collaboration aims to expedite the development of GS-100, a promising gene therapy for NGLY1 Deficiency, through crucial Phase I/II/III trials.
Image Source : Public Domain

Image Source : Public Domain

 Andelyn 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.


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