Allergan and NIBRT announce gene therapy research collaboration

The National Institute for Bioprocessing Research and Training (NIBRT) collaboration is part of Allergan’s Open Science strategy and will address key challenges in the manufacture and characterisation of Adeno-Associated Virus (AAV)-based gene therapies.

Adeno-Associated virus (AAV)-based gene therapies represent an exciting new wave of advanced therapy medicinal products (ATMPs) that are being generated for the treatment of ailments caused by a defective gene within certain patient populations. AAV based gene therapy is used to deliver a correctly functioning copy of the gene to the patient. Very promising results have been reported in genetic diseases including haemophilia and ocular 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 is challenging. Furthermore, ensuring the quality and efficacy of the AAV material also requires technology development. This collaboration between NIBRT and Allergan Biologics will address the following challenges in AAV production:

• Optimising cell culture process for generation of infectious AAV particles of various serotypes
• Establishing vectors and transfection/transduction conditions to minimise incorrect packaging events
• Investigation of sensitive analytical methods for the characterisation of the resulting AAV particles that enable differentiation between full and empty AAV capsids

Commenting on the collaboration Dr Crawford Brown, SVP Biologics & Small Molecule (API), Allergan remarked:  “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.  This is part of Allergan’s Open Science strategy which focuses on leveraging next generation technology to benefit our patients”.

Prof Niall Barron, who will lead the project at NIBRT with Prof Jonathan Bones, welcomed the innovative project: “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.”  

National Institute for Bioprocessing Research and Training (NIBRT)

The National Institute for Bioprocessing Research and Training (NIBRT) is a global centre of excellence for training and research in biopharmaceutical manufacturing. NIBRT is located in a world class facility in Dublin, Ireland. This facility is purpose built to closely replicate a modern bioprocessing plant with state-of the art equipment and enables NIBRT to offer the highest quality training and research solutions. NIBRT’s mission is to support the growth and development of all aspects of the biopharmaceutical manufacturing industry.