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

In a significant collaboration aimed at combating a life-threatening illness, Andelyn Biosciences, a leading cell and gene therapy organization, has joined forces with Grace Science, a biotech firm. Their joint mission is to facilitate the transfer of technology and production of GS-100, a gene therapy utilizing a suspension process AAV for NGLY1 Deficiency. This rare and severe condition currently lacks an approved treatment, causing lifelong suffering with symptoms such as developmental delays and cognitive impairments.

GS-100, based on a recombinant AAV9 vector, carries the complete human NGLY1 gene. It has received the prestigious orphan drug designation from both the FDA and EMA, highlighting its importance for treating rare diseases. The therapy also boasts a Rare Pediatric Disease Designation, which could lead to a Priority Review Voucher and a Fast-Track designation. Grace Science has already administered GS-100 to the first patient with NGLY1 Deficiency and is set to treat a second patient in the coming months.

This partnership will harness Andelyn's extensive knowledge in AAV processes, particularly in the later stages of manufacturing and getting the product ready for the market. It is designed to ensure quality while speeding up the production schedule, bringing much-needed hope to those affected by NGLY1 Deficiency.

Andelyn Biosciences' Chief Commercial Officer, Matt Niloff, expressed the company's honor in collaborating with Grace Science, praising their commitment to addressing rare diseases. He emphasized the shared values of collaboration, customer focus, and a dedication to quality, which will bolster confidence in the success of the GS-100 program.

Matt Wilsey, CEO and Co-Founder of Grace Science, echoed the enthusiasm for the partnership, noting that Andelyn's expertise in AAV gene therapy manufacturing is crucial for providing NGLY1 patients with a safe and high-quality drug for clinical trials and future use.

Andelyn Biosciences, with over two decades of experience, is dedicated to advancing discoveries in cell and gene therapies for both rare and common diseases. The company's comprehensive services include technology transfer, analytical process development, and high-quality manufacturing of vectors and plasmids at its facilities in Columbus, Ohio.

Grace Science, established in 2017 by Matt Wilsey and Dr. Carolyn Bertozzi, is focused on developing innovative therapies based on the NGLY1 enzyme's role in proteostasis. Their in-depth understanding of the NGLY1 pathway is instrumental in finding new treatments for NGLY1 Deficiency and other diseases.