Monday, March 25, 2024
Andelyn Biosciences, Inc., a leading Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy, has partnered with Grace Science, LLC, to facilitate the tech transfer and production of GS-100, an AAV NGLY1 gene therapy in suspension form, for Phase I/II/III clinical trials aimed at treating NGLY1 Deficiency. This collaboration aims to enhance program efficiency, support ongoing clinical trials, and expedite the delivery of this crucial therapy to patients.
NGLY1 Deficiency is a severe and life-threatening condition without an approved treatment, causing individuals to endure a range of debilitating symptoms including developmental delays, cognitive impairment, (hypo) alacrimia, movement disorders, and other neurological issues. GS-100, a recombinant AAV9 vector encoding the full-length human NGLY1 gene, received orphan drug designation (ODD) from both the FDA and the European Medicine Agency (EMA) in 2021. Additionally, it was granted Rare Pediatric Disease Designation by the FDA the same year, potentially qualifying for a Priority Review Voucher upon marketing approval and receiving Fast-Track designation in 2023. Grace Science administered the first dose of GS-100 to an NGLY1 Deficiency patient in February of this year and plans to dose the second patient in May 2024.
In this collaboration, Grace Science will benefit from Andelyn's expertise in AAV processes and their capabilities in late-stage manufacturing and commercial readiness. The partnership emphasizes a commitment to quality while also expediting Grace's manufacturing timelines, providing hope to patients affected by NGLY1 Deficiency.
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed his gratitude for the opportunity to collaborate with Grace Science, highlighting the latter's dedication to addressing rare diseases. Both companies prioritize collaboration and uphold a customer-centric approach and a commitment to quality, instilling confidence and hope in patients and their families regarding the success of the GS-100 program.
Matt Wilsey, CEO and Co-Founder of Grace Science, shared his enthusiasm for the partnership, emphasizing Andelyn's extensive experience and expertise in AAV gene therapy manufacturing. He emphasized that Andelyn's involvement ensures NGLY1 patients have access to safe, high-quality drug products for the clinical trial and beyond.
Andelyn Biosciences is dedicated to advancing client discoveries and manufacturing life-changing cell and gene therapies for both rare and prevalent diseases. With a foundation built on extensive AAV development and manufacturing experience, Andelyn offers technology transfer capabilities, data-driven analytical-based process development, and quality vector and plasmid manufacturing across its three facilities in Columbus, Ohio.
Source: prnewswire.com
