Andelyn Biosciences Inc, a cell and gene therapy Contract Development and Manufacturing Organisation (CDMO), announced on Tuesday that it has partnered with Queen's University to develop and manufacture AAV9-GM2, a gene therapy candidate for the treatment of GM2 gangliosidoses, such as Tay-Sachs and Sandhoff diseases.

GM2 gangliosidosis is a rare, inherited disorder that progressively destroys nerve cells in the brain and spinal cord. While the most common form presents in infancy, other forms may emerge in childhood, adolescence, or adulthood. As a lysosomal storage disorder (LSD), GM2 is caused by genetic mutations that disrupt normal lysosomal function, leading to the accumulation of harmful substances in cells and severe neurological decline.

Under the partnership, Andelyn is leveraging its AAV Curator Platform to execute a multi-phase programme to advance AAV9-GM2 for dosing patients. The AAV Curator Platform is Andelyn's regulatory-proven viral vector process, complete with a cell line and a modular approach to adapting unit operations to program requirements.