- Andelyn Biosciences has partnered with Queen’s University to develop and manufacture AAV9-GM2, a gene therapy candidate for GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases.
- The company will use its AAV Curator Platform to support a multi-phase program aimed at advancing AAV9-GM2 toward patient dosing.
Andelyn Biosciences has partnered with Queen’s University to develop and manufacture AAV9-GM2, a gene therapy candidate being developed for GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. The partnership will support the advancement of the candidate toward clinical dosing.
Andelyn will use its AAV Curator Platform, a viral vector manufacturing process that includes a cell line and a modular approach for adapting unit operations to program requirements. The company said the platform is intended to support yield and quality as the AAV9-GM2 program progresses.
GM2 gangliosidoses are rare inherited disorders that cause progressive damage to nerve cells in the brain and spinal cord. The conditions are classified as lysosomal storage disorders and result from genetic mutations that disrupt normal lysosomal function.
Under the partnership, Andelyn will provide its AAV development and production capabilities to support Queen’s University’s program. The company said its role will include manufacturing the viral vector required for the upcoming GM2 program.
“We are proud to partner with Queen’s University to advance this gene therapy candidate for the benefit of patients and families facing the challenges of GM2 gangliosidoses. Our deep expertise in AAV development and production allows us to support Queen’s University with the scale and quality rigor necessary to bring this life-changing therapy one step closer to reality.”
Matt Niloff, Chief Commercial Officer at Andelyn Biosciences
