Andelyn Biosciences is set to develop and manufacture a gene therapy known as AAV9-GM2, for Queen’s University to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. Andelyn Biosciences is set to develop and manufacture a gene therapy known as AAV9-GM2, for Queen’s University to treat GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. Using its platform, Andelyn Biosciences plans to implement a multi-phase programme. Credit: Gorodenkoff / Shutterstock.com. ... The partnership aims to progress this gene therapy using Andelyn’s AAV Curator Platform. GM2 gangliosidosis is a rare genetic disorder that leads to the progressive loss of nerve cells in the brain and spinal cord. The condition belongs to the group of lysosomal storage disorders and results from genetic mutations that impair lysosomal function, leading to harmful accumulation within cells and severe neurological symptoms. Andelyn Biosciences will implement a multi-phase programme using its Curator Platform, which features a cell line and a modular approach intended to adapt manufacturing processes to specific programme needs. Queen’s University medical geneticist and department of paediatrics professor Dr Jagdeep Walia said: “This partnership with Andelyn is very strategic as they have the established expertise of producing vectors for many gene therapy programmes. In August 2025, Andelyn Biosciences entered a collaboration with Amplo Biotechnology to manufacture adeno-associated virus (AAV) gene therapies to address conditions affecting the neuromuscular junction.

18, 2026 /PRNewswire/ -- Andelyn ... cell and gene therapy Contract Development and Manufacturing Organization (CDMO), 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 ... 18, 2026 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), 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. This partnership reflects Andelyn's continued commitment to supporting innovative gene therapy programs targeting rare and ultra-rare diseases, where speed, quality, and manufacturing expertise are critical to advancing therapies to patients. ... Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization, and production of viral vectors for gene therapy. 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. Operating out of its development and manufacturing facilities in Columbus, Ohio, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing.

18, 2026 /PRNewswire/ -- Andelyn ... cell and gene therapy Contract Development and Manufacturing Organization (CDMO), 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 ... 18, 2026 /PRNewswire/ -- Andelyn Biosciences, Inc., a leading patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), 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. This partnership reflects Andelyn's continued commitment to supporting innovative gene therapy programs targeting rare and ultra-rare diseases, where speed, quality, and manufacturing expertise are critical to advancing therapies to patients. ... Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization, and production of viral vectors for gene therapy. 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. Operating out of its development and manufacturing facilities in Columbus, Ohio, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing.

A recent study explores adult-onset Sandhoff disease, revealing a motor neuron disease phenotype through patient-derived models. This research provides new mechanistic insights that could inform future therapeutic strategies.

Researchers have successfully generated and characterized induced pluripotent stem cell lines from patients with Tay-Sachs and Sandhoff disease. This advancement may facilitate further studies into the pathophysiology and potential therapies for these lysosomal storage disorders.