Singapore, Aug. 19 -- Andelyn Biosciences has partnered with Queen's University to develop and manufacture AAV9-GM2, an investigational gene therapy for GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases.

GM2 gangliosidoses are rare inherited lysosomal storage disorders caused by genetic mutations that disrupt normal lysosomal function. The resulting accumulation of harmful substances in cells leads to progressive damage to nerve cells in the brain and spinal cord.

The condition most commonly presents during infancy, although later-onset forms can emerge during childhood, adolescence or adulthood.

Under the partnership, Andelyn Biosciences will use its AAV Curator Platform in a multi-phase programme intended to advance AAV9-...