Andelyn Biosciences and Queen’s University have partnered to develop and manufacture AAV9-GM2, a gene therapy candidate for GM2 gangliosidoses.

AAV9-GM2 programme targets rare neurological diseases

The partnership will use Andelyn’s AAV Curator platform to support development and manufacturing of AAV9-GM2, a gene therapy candidate being developed for GM2 gangliosidoses including Tay-Sachs and Sandhoff diseases.

GM2 gangliosidoses are inherited lysosomal storage disorders caused by genetic mutations that impair the breakdown of certain substances within cells. The resulting accumulation can progressively damage nerve cells in the brain and spinal cord.

The diseases can present at different ages, with the most severe forms beginning during infancy and other forms developing later in childhood, adolescence or adulthood.

Under the partnership, Andelyn will provide viral vector development and manufacturing capabilities as the AAV9-GM2 programme progresses towards clinical development.

Matt Niloff, chief commercial officer at Andelyn Biosciences, 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.”

Manufacturing programme uses AAV9 platform

Andelyn will use its AAV Curator platform to carry out a multi-phase development and manufacturing programme for AAV9-GM2.

The platform uses a cell line and modular manufacturing processes that can be adapted according to programme requirements.

The company said the approach is intended to support viral vector production as AAV9-GM2 advances towards potential clinical use.

AAV vectors are widely used in gene therapy because they can deliver genetic material to target cells. AAV9 is one of several serotypes being investigated for delivery of genetic material to the nervous system.

The partnership therefore combines Queen’s University research and clinical expertise in GM2 gangliosidoses with Andelyn’s viral vector development and manufacturing capabilities.

Supporting development of a rare disease therapy

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.”

Walia added that Andelyn’s manufacturing experience would support production of the viral vector for the planned GM2 programme.

The collaboration is focused on advancing AAV9-GM2 towards patient dosing. The announcement does not indicate that clinical dosing has begun.

GM2 gangliosidoses currently have limited treatment options and are associated with progressive neurological deterioration, creating a need for approaches that address the underlying genetic cause.

The partnership adds a manufacturing component to the development of AAV9-GM2 and will support its progression through the stages required before potential clinical use.