Going Viral: Adeno-Associated Virus Therapeutics

KTN and MedImmune hosted the Development of AAV Therapeutics event in January 2019.

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MedImmune and KTN organised the event, ‘Development of AAV Therapeutics’, which was held on 30th January at MedImmune’s Cambridge site. The meeting brought together professionals from¬†industry and academia to disseminate and share information on the development of adeno-associated virus therapeutics (AAV), specifically focussing on manufacturing and highlighting the areas of need for this community.

Buoyed by the recent approval of two AAV-based gene therapy products, AAV has emerged as a popular choice for the development of gene therapies. The event welcomed over 90 delegates from 40 organisations covering the whole supply chain, including companies, universities, consultancies and RTOs.

For AAV to realise its full potential and the UK to capitalise on its strengths in the field, various challenges were highlighted by presenters. Some of the challenges raised included:

• Viruses, such as AAV, are complex and there’s a necessity to carry out a carefully choreographed set of steps in order to transfer genes successfully. In order to use them as effective therapeutic vectors, we first need to have a good understanding of their biology.
• The intellectual property landscape regarding AAV capsids is complex.
• AAV process yield needs improvement, and bioprocessing and analytics require standardisation.
• Contrary to protein-based biotherapeutics, the US Food and Drug Administration (FDA) considers the CMC risk for gene therapies to be higher than the clinical risk.
• Currently, AAV therapeutics are developed for a limited set of diseases (many of which are orphan diseases).
• Clinical AAV gene therapy studies often target a small number of patients, for whom it may be challenging to find affordable GMP manufacturing.
• Capacity and capability remain a challenge globally, and the UK has work to do to stay at the forefront of this sector.
• Skillsets and people are key to the growth of this sector.

Importantly, these challenges offer opportunities to innovate, and thus to grow the UK supply chain and the economy. Delegates remarked that this was the first time that this particular community, who have a shared focus on AAV, had gathered and there was interest to see the wider awareness of the landscape. In addition to the research and clinical excellence from world-class UK universities, there are a number of nascent and growing SMEs and larger biopharma companies working on AAV therapeutics, as well as CDMOs and consultancies. In order to boost innovation, the sector is supported by Research and Technology Organisations such as the Cell & Gene Therapy Catapult and the CPI National Biologics Manufacturing Centre. There have additionally been significant investments through the Industrial Strategy Challenge Fund, both in infrastructure and collaborative R&D, to support the wider ATMP field.

There was a lot of energy in the room on the day where people from different organisations were able to exchange ideas, network and discuss working collaboratively, therefore playing a role in supporting the success of the AAV community in the UK. Lekan Daramola, Associate Director at MedImmune commented, “We welcomed the opportunity to work with the Knowledge Transfer Network in order to organise and deliver this event. KTN were key to developing an excellent programme and attract relevant delegates, which resulted in a high level of engagement during the meeting. We are looking forward to working with them on future events.”

KTN is on hand to support companies wishing to explore wider collaborations, connections or funding to support innovation. The Biomedical Catalyst is one such opportunity this year. Please get in touch with Marcel Kuiper or Sarah Goulding if you wish to discuss a project or proposal idea.