The CGT specialist Andelyn Biosciences is partnering with Queen’s University in Belfast to develop and manufacture a gene therapy candidate for the treatment of GM2 gangliosidoses, such as Tay-Sachs and Sandhoff diseases.
Andelyn will support Queen's Belfast in producing and advancing AAV9-GM2, using its AAV Curator Platform to execute a multi-phase programme to bring the candidate to patient dosing.
The AAV Curator Platform is a viral vector process, complete with a cell line and a modular approach to adapting unit operations to programme requirements.
Andelyn says the platform delivers both yield and quality.
“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 rigour necessary to bring this life-changing therapy one step closer to reality.
Dr Jagdeep Walia, Medical Geneticist and Professor in the Department of Paediatrics at Queen’s University, added: “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.
