The future of gene therapies: three insights from the FORUM workshop

On the 7 March 2019, the Academy and the Cell and Gene Therapy Catapult hosted a FORUM workshop on ‘The future of gene therapies: next steps for the UK’. The workshop brought together a diverse range of speakers and stakeholders from across academia, industry, the NHS, charities, funding bodies and regulators, among others, with the aim of highlighting the challenges facing the translation of gene therapy research and how the U.K can become a world-leading environment for the development of gene therapies.

A summary report of proceedings will be published on the Academy’s website later this year. Until then, here are three key lessons we learnt about the future development of gene therapies.

1. Persistence is key to success

Remarking on the perceived ‘failures’ of gene therapy technology, the speakers reflected on the significant challenge that developing gene therapies has been, but noted that the field was finally beginning to mature to the point where there are many promising therapies that are likely to be available for patients over the coming years. Participants pointed to the delay between the first gene therapy clinical trial in 1990 and the first EU-approved gene therapy in 2012. Despite setbacks as a field over the last 30 years, participants emphasised the need for persistence in the face of setbacks in order to deliver the life transforming medicines to patients whose needs are not met through conventional medicines. However participants warned against unwarranted hype; noting that a lot of research still needs to be done before gene therapies form a standard part of NHS care.   

2. Proof of concept, but many hurdles remain

The successful deployment of gene therapies is reliant on being able to deliver personalised medicine within the healthcare system. In the case of gene therapies, participants pointed out the many challenges facing the adoption of gene therapies into the healthcare system; including building effective manufacturing and supply chains to allow for small scale medicines to be delivered and reimbursing therapies that may have high upfront costs but have the potential to provide long term benefits for diseases that have few other treatment options.  

3. Supportive networks are crucial

Many of the speakers extolled the benefits of having a closer working relationship between researchers, both within industry and academia, and funders and regulators. It was emphasised that it is helpful for researchers to approach funding bodies early on to identify the potential funding streams available, to provide advice on potential issues their studies may face and how they might be rectified to ensure that project milestones are achieved. Similarly, regulatory bodies such as the Medicines and Healthcare products Regulatory agency can provide early advice to help researchers and companies navigate regulation efficiently.

These three highlights offer a small snapshot into the wealth of discussion at this workshop. They highlight both the opportunity of gene therapy technology to bring lifelong benefits to patients and the challenges that the technology and researchers must face so that gene therapy ban be brought forward to mainstream consumption. Closer collaboration between different sectors will help the innovation required to address the issues facing gene therapy technologies.

This workshop was by invitation only. To discuss this workshop further please contact forum@acmedsci.ac.uk