Andelyn to Manufacture Queen’s AAV9-GM2 Gene Therapy

Andelyn Biosciences has been selected to develop and manufacture AAV9-GM2, an experimental gene therapy for GM2 gangliosidoses (including Tay-Sachs and Sandhoff diseases), on behalf of Canada’s Queen’s University. Announced on 19 August 2026, the multi-phase partnership will use Andelyn’s proprietary AAV Curator Platform, targeting a rare disease category with no approved therapies and substantial unmet clinical need for progressive neurodegeneration.

GM2 gangliosidosis is a rare genetic disorder in the lysosomal storage disorder family, caused by mutations that impair lysosomal function and drive harmful accumulation within cells. The condition results in progressive loss of nerve cells in the brain and spinal cord and severe neurological symptoms, with the most common form beginning in infancy. The programme will use Andelyn’s AAV Curator Platform, which features a proprietary cell line and modular design intended to adapt manufacturing processes to specific programme needs. Dr Jagdeep Walia, medical geneticist and paediatrics professor at Queen’s University, positioned the partnership as strategically important, citing Andelyn’s established viral vector production expertise.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, positioned the partnership as consistent with the company’s focus on advancing gene therapy candidates through deep AAV development and production expertise. The partnership builds on Andelyn’s August 2025 collaboration with Amplo Biotechnology to manufacture AAV gene therapies for neuromuscular junction conditions. Andelyn competes with Catalent, Lonza, Thermo Fisher’s Patheon, Fujifilm Diosynth Biotechnologies and Samsung Biologics across the AAV gene therapy manufacturing category, while the disease landscape includes gene therapy developers Taysha Gene Therapies, Passage Bio and REGENXBIO.

The commercial signal is a rare disease gene therapy CDMO category continuing to expand as academic and biotech developers outsource complex AAV manufacturing. Andelyn’s focus on lysosomal storage disorders extends its rare disease footprint. Expect Catalent, Lonza, Fujifilm Diosynth, Thermo Fisher’s Patheon and Samsung Biologics to compete aggressively for similar academic gene therapy partnerships through 2027 as the AAV CDMO market matures.

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