Компания Andelyn Biosciences и Университет Квинса запустят производство генной терапии для лечения болезней ГМ2

Andelyn Biosciences to implement Curator Platform to develop and manufacture gene therapy AAV9-GM2 Andelyn is leveraging its AAV Curator Platform to execute a multi-phase program to advance AAV9-GM2 for dosing patients 27 Aug 2026 Andelyn Biosciences, Inc. 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 program to advance AAV9-GM2 for dosing patients. The AAV Curator® Platform is Andelyn’s long-standing, regulatory-proven viral vector process, complete with a cell line and a modular approach to adapting unit operations to program requirements. With this methodology, both yield and quality are delivered. “We are proud to partner with Queen’s University to advance this gene therapy candidate for the benefit of patients and families facing the challenges of GM2 gangliosidoses,” said Matt Niloff, Chief Commercial Officer at Andelyn Biosciences. “Our deep expertise in AAV development and production allows us to support Queen’s University with the scale and quality rigor necessary to bring this life-changing therapy one step closer to reality.” Dr. Jagdeep Walia, medical geneticist and Professor in the Department of Pediatrics at Queen’s University, said, “This partnership with Andelyn is very strategic as they have the established expertise of producing vectors for many gene therapy programs. Their track record and ability to manufacture the viral vector for the upcoming GM2 program gives us confidence that the participants in the clinical trial will be receiving the highest quality, safe product.” Want the latest science news straight to your inbox? Become a SelectScience member for free today>> Tags Frequently asked questions Show frequently asked questions What is AAV9-GM2, and which diseases is it designed to treat? AAV9-GM2 is a gene therapy candidate being developed and manufactured by Andelyn Biosciences and Queen’s University. It is intended to treat GM2 gangliosidoses, including Tay-Sachs disease and Sandhoff disease. How does GM2 gangliosidosis affect the brain and spinal cord? GM2 gangliosidosis is a rare inherited lysosomal storage disorder caused by genetic mutations that disrupt lysosomal function. Harmful substances accumulate in cells, progressively destroying nerve cells in the brain and spinal cord and causing severe neurological decline. How is Andelyn Biosciences supporting the AAV9-GM2 gene therapy program? Andelyn Biosciences is using its regulatory-proven AAV Curator® Platform in a multi-phase program to advance AAV9-GM2 toward patient dosing. The platform combines a cell line with modular unit operations to meet program requirements while delivering vector yield, quality, and manufacturing scale.