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.

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 most common form begins during infancy, although other forms can appear later in life.

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.

Andelyn Biosciences chief commercial officer Matt Niloff said: “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.

“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.”

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.

“Their track record and ability to manufacture the viral vector for the upcoming GM2 programme gives us confidence that the participants in the clinical trial will be receiving the highest quality, safe product.”

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.