Gene therapy has been an area with a growing level of commercial interest in recent years, with a wide range of licensing deals and funding rounds reported.

The latest of these is a new licensing agreement reached between King’s College London and Pfizer for the development of a series of adeno-associated virus (AAV) gene therapy vectors.

There are various technical approaches for delivering a therapeutic gene, but using viral vectors is the most commonly used in commercial development.  Of the viral vectors currently available, AAV is the most common currently in use.

The project is the work of Professor Michael Linden and Dr Els Henckaerta from the Department of Infectious Diseases, and has been developed with help from the King’s Commercialisation Institute.

The team have developed an AAV vector platform that’s based on the discovery that a series of targeted capsid mutations in AAV vectors yields a new generation of vectors with superior transduction abilities in the brain. The agreement gives Pfizer the exclusive right to use this platform.

The license will guarantee an upfront payment for King’s College London, with additional payments possible after clinical development and commercialization milestones are reached.

Pfizer will also fund further research into the AAV platform and its application in gene therapy via it’s Rare Disease Consortium (RDC).  They will also fund new research into furthering our basic understanding of the virus to help ensure it’s fit for clinical use.

Dr Els Henckaerts from the Division of Immunology, Infection & Inflammatory Disease at King’s College London, said: “This is a very exciting moment, emerging from several years of basic science discovery and translational research in my laboratory. The collaboration with Pfizer provides a very strong platform for us to work toward progressing this potential new therapy into full clinical development for the benefit of patients with rare diseases.”