Pharma Pioneer

Andelyn and Grace Science Collaborate on AAV Gene Therapy Manufacturing for NGLY1 Deficiency

19 May 2024
3 min read

In a collaborative effort to address NGLY1 Deficiency, a severe and life-threatening condition, Andelyn Biosciences has teamed up with Grace Science, LLC, to facilitate the technical transfer and production of GS-100, an AAV NGLY1 gene therapy for clinical trials. This gene therapy is designed to treat patients who currently have no approved treatment options and experience a range of debilitating symptoms such as developmental delays and cognitive impairments.
GS-100 utilizes a recombinant AAV9 vector to deliver the full-length NGLY1 gene. It has received significant regulatory support, including orphan drug designation from both the FDA and EMA, and a Rare Pediatric Disease Designation from the FDA, which could lead to a Priority Review Voucher and Fast-Track status. The first patient was dosed in February, with a second dosing planned for May 2024.
This partnership aims to enhance the efficiency of the program and expedite the availability of this vital treatment. Grace Science will utilize Andelyn's advanced AAV process knowledge and manufacturing capabilities to ensure a high-quality product for clinical trials. The collaboration emphasizes a commitment to quality and efficiency, which is expected to benefit patients and their families.
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed the company's honor in working with Grace Science, highlighting their shared values of collaboration, customer focus, and quality. Matt Wilsey, CEO of Grace Science, echoed the enthusiasm for the partnership, noting Andelyn's expertise in AAV manufacturing as a key factor in providing a safe and high-quality drug product for their clinical trial.
Andelyn Biosciences, with over two decades of experience in AAV development, offers comprehensive services from concept to commercial manufacturing. The company operates from three facilities in Columbus, Ohio, and has a track record of producing cGMP materials for numerous clinical batches and trials. Andelyn's services include technology transfer, process development, and manufacturing of both adherent and suspension processes, with a capacity of up to 2,000 liters.
Grace Science, established in 2017 by Matt Wilsey and Dr. Carolyn Bertozzi, is focused on developing therapies for NGLY1-related conditions. The company's expertise in the NGLY1 pathway is central to its mission of creating novel treatments for both rare and common diseases.

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