Andelyn Biosciences Inc., a leading cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has recently partnered with Grace Science LLC to transfer technology and manufacture GS-100. GS-100 is a suspension process AAV NGLY1 gene therapy intended for Phase I/II/III clinical trial material for the treatment of NGLY1 Deficiency.
NGLY1 Deficiency is a severe, life-threatening disease that currently has no approved therapy. Individuals with this condition endure debilitating symptoms throughout their lives. The collaboration between Andelyn Biosciences and Grace Science aims to improve efficiency, support ongoing clinical trials, and expedite the delivery of this crucial therapy to patients.
GS-100 is a recombinant AAV9 vector that contains a full-length version of the human NGLY1 gene. In 2021, it received orphan drug designation (ODD) from the FDA and the European Medicine Agency (EMA). Additionally, in 2021, the FDA granted GS-100 Rare Pediatric Disease Designation. These designations open up possibilities 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, and plans to dose the second patient in May 2024.
Through this partnership, 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 gratitude for this opportunity to collaborate with Grace Science. He emphasized shared values of strong collaboration, customer focus, and commitment to quality.
Matt Wilsey, CEO and Co-Founder of Grace Science expressed his excitement about this partnership with Andelyn Biosciences. He highlighted Andelyn’s expertise in AAV gene therapy manufacturing will ensure that NGLY1 patients have access to a safe high-quality drug product for clinical trial use.
This partnership instills confidence and hope in patients and their families regarding the success of the GS-100 program.
In conclusion, this partnership between Andelyn Biosciences Inc., a leading CDMO specialized in cell & gene therapies manufacturing processes & services;
