What is the future of gene therapies?

Gene therapies have huge potential to address unmet needs for a variety of diseases, but collaboration will be key to unlocking this potential, according to our latest FORUM report.

In March 2019, the Academy held a one day workshop in partnership with the Cell and Gene Therapy Catapult that explored how the UK could remain at the forefront of gene therapy development in the context of recent advances within the sector.

Co-chaired by Dr Melanie Lee CBE FMedSci, Chief Executive Officer at LifeArc and Professor Uta Griesenbach, Professor of Molecular Medicine at  Imperial College London, the workshop brought together representatives from across the advanced therapies sector to discuss the translational challenges facing the next generation of gene therapies and what practical steps can be taken to overcome them.

Several major themes emerged from the workshop, with key focus being given to stabilising the route of gene therapies from research in academia through to deliverable and effective therapies. Building underlying supportive and collaborative networks across the gene therapy landscape would ensure that the UK remains at the forefront of this emerging technology.

Supporting the rich source of gene therapy research from academia was seen as crucial in starting any progress developing such therapies, designing mechanisms to secure funding for this research and help it navigate the pathway from research to manufacture. Attracting increased funding through venture capitalist investment and the development of new reimbursement models for novel gene therapies would help to create the stability required for the expansion of gene therapy research and the development and adoption of new therapies.

Initiatives such as the Advanced Therapy Treatment Centres and the Cell and Gene Therapy Catapult facilities were noted for helping to support localised discovery, manufacture and delivery but more supportive networks will be needed to support discovery research programmes. 

Both chairs noted that bringing the community together was a positive step and had brought more clarity to the challenges that gene cell therapies faced. It was clear to the chairs that the UK was at the ‘forefront’ of gene therapy development and overcoming the challenges identified by the attendees would allow the UK to remain there.

The full report from the workshop is now available to view and download online.

The FORUM is the Academy’s neutral and independent platform for individuals across academia, industry, the NHS, Government, charity, regulatory and the wider healthcare sector to meet and take forward national discussions on scientific opportunities.

Find out more about the FORUM on our website.