Gene therapy treatments for a number of common hereditary diseases are to be the focus of a new partnership between industry and researchers. Multinational pharma giant Allergan and the National Institute for Bioprocessing Research and Training (NIBRT) announced the partnership, saying it will focus on gene therapies developed from adeno-associated virus (AAV).

AAV-based gene therapy is used to deliver a correctly functioning copy of the gene to the patient. So far, it has reported promising results in genetic diseases including haemophilia and macular degeneration with a growing number of gene therapy products now approved.

However, current yields of AAV from the manufacturing process mean that supplying patients with the required quantities of medicine has proven a challenge, as well as ensuring its quality and efficacy.
In a joint statement, Allergan and NIBRT said that this partnership will have a number of objectives including optimising the cell culture process of infectious AAV particles of various serotypes, establishing transfection/transduction conditions to minimise incorrect packaging events, and investigating sensitive analytical methods that can help differentiate between full and empty AAV capsids.

‘Potentially transformative’
Commenting on the collaboration, Dr Crawford Brown, senior vice-president of biologics and small molecule at Allergan, said: “We are very excited to work with NIBRT on this potentially transformative gene therapy project.

“Given the potential for gene therapy to transform patients’ lives, this collaboration will help progress both the analytics and process, which are currently the main challenges in the production of high quality AAV medicines.”

Prof Niall Barron of NIBRT – who will lead the project along with Prof Jonathan Bones – added: “The collaboration with Allergan focuses on some of the very real and immediate challenges to ensuring ground-breaking new gene therapies are safely and successfully delivered to patients in need.

“NIBRT researchers will combine expertise gained from many years of experience in the recombinant protein space with complimentary expertise in Allergan to improve AAV-based gene therapy production.”

Colm Gorey

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