Andelyn Biosciences to manufacture AAV9-GM2 gene therapy for GM2 gangliosidoses
GM2 gangliosidoses are a group of rare inherited disorders that cause the progressive loss of nerve cells in the brain and spinal cord.
USA—Andelyn Biosciences will develop and manufacture AAV9-GM2, an investigational gene therapy being advanced by Queen’s University to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases.
The partnership will use Andelyn’s AAV Curator Platform to support the development and manufacturing of the gene therapy as it progresses towards clinical testing.
Targeting rare genetic disorders
GM2 gangliosidoses are a group of rare inherited disorders that cause the progressive loss of nerve cells in the brain and spinal cord.
Tay-Sachs and Sandhoff diseases are among the most severe forms of the condition, with symptoms often beginning during infancy, although later-onset forms can also occur.
The disorders belong to a broader group of diseases known as lysosomal storage disorders.
Genetic mutations impair the normal function of lysosomes, cellular structures responsible for breaking down and recycling various substances.
As a result, harmful materials accumulate inside cells, particularly in nerve cells, leading to progressive neurological impairment.
AAV9-GM2 is being developed as a potential treatment for these disorders by using an adeno-associated virus (AAV) vector to deliver genetic material intended to address the underlying cause of the disease.
Manufacturing partnership
Under the agreement, Andelyn Biosciences will implement a multi-phase development and manufacturing programme using its AAV Curator Platform.
The platform combines a cell line with a modular manufacturing approach designed to allow processes to be adapted according to the requirements of individual gene therapy programmes.
The company said the approach will support the development of the AAV9-GM2 programme while maintaining the manufacturing standards required for clinical development.
Matt Niloff, chief commercial officer at Andelyn Biosciences, said the company was proud to partner with Queen’s University to advance the gene therapy candidate for patients and families affected by GM2 gangliosidoses.
“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,” Niloff said.
Dr Jagdeep Walia, a medical geneticist and professor in the Department of Paediatrics at Queen’s University, described the partnership as strategic because of Andelyn’s established experience in producing viral vectors for gene therapy programmes.
He said the company’s track record and ability to manufacture the viral vector for the upcoming GM2 programme give the research team confidence that participants in the clinical trial will receive a high-quality and safe product.
Expanding AAV manufacturing activities
The agreement adds to Andelyn Biosciences’ work in developing and manufacturing AAV-based therapies.
In August 2025, the company entered into a collaboration with Amplo Biotechnology to manufacture AAV gene therapies targeting conditions affecting the neuromuscular junction.
Through the latest partnership, Andelyn will provide its AAV development and manufacturing capabilities to support Queen’s University as AAV9-GM2 advances through its development programme.
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