Andelyn Biosciences will develop and manufacture an AAV9 gene therapy for GM2 gangliosidosis — a group of rare, fatal inherited disorders that includes Tay-Sachs and Sandhoff diseases — in partnership with Queen’s University.
GM2 gangliosidosis is a lysosomal storage disorder in which a missing enzyme lets toxic material build up in nerve cells, causing progressive loss of brain and spinal-cord neurons. There are no approved disease-modifying treatments for most forms. Andelyn will produce the therapy using its AAV Curator Platform, a modular manufacturing approach with a dedicated cell line and flexible processes.
Where it stands
The program is in preclinical development, with clinical trials planned; Andelyn will oversee a multi-phase development effort. “Our deep expertise in AAV development and production allows us to support Queen’s University with the scale and quality rigour necessary to bring this life-changing therapy one step closer to reality,” said Andelyn CCO Matt Niloff. Queen’s University’s Dr. Jagdeep Walia said the manufacturer’s track record gives confidence that trial participants “will be receiving the highest quality, safe product.”
Why it matters
For fatal pediatric neurodegenerative diseases with no good options, reliable, high-quality manufacturing of a viral-vector gene therapy is a critical step — often the difference between a promising academic program and one that can actually reach patients in a trial.