Andelyn Biosciences and Grace Science LLC Partner to Manufacture GS-100 AAV Gene Therapy for NGLY1 Deficiency Patients

Andelyn Biosciences Collaborates with Grace Science to Further Develop GS-100

Grace Science LLC and Andelyn Biosciences Inc., a cell and gene therapy Contract Development and Manufacturing Organization (CDMO), have formed a partnership to tech transfer and manufacture GS-100. GS-100 is a suspension process AAV NGLY1 gene therapy that aims to treat NGLY1 Deficiency in Phase I/II/III clinical trials.

NGLY1 Deficiency is a severe, life-threatening disease with no approved therapy. Patients with this condition suffer debilitating symptoms throughout their lives. The collaboration between the two companies aims to improve efficiency, support ongoing clinical trials, and expedite the delivery of this crucial therapy to patients.

In 2021, GS-100 received orphan drug designation (ODD) from the FDA and the European Medicine Agency (EMA). Additionally, the FDA granted GS-100 Rare Pediatric Disease Designation in 2021. These designations open up the possibility for Priority Review Voucher upon marketing approval and Fast-Track designation in 2023. Grace Science successfully administered the first dose of GS-100 to an NGLY1 Deficiency patient in February 2024, with plans to dose the second patient in May 2024.

The partnership between Andelyn Biosciences and Grace Science will utilize Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness. This collaboration accelerates Grace’s manufacturing timelines, offering hope to individuals suffering from NGLY1 Deficiency.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed excitement about collaborating with Grace Science and emphasized shared values of strong collaboration, customer-centric focus, and commitment to quality. This partnership instills 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, conveyed enthusiasm about partnering with Andelyn Biosciences due to its expertise in AAV gene therapy manufacturing. He emphasized that Andelyn’s capabilities will ensure that NGLY1 patients have access to a safe, high-quality drug product for clinical trials purposes as well as beyond.

Overall, this collaboration will improve efficiency

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