3 questions with…
Professor of Paediatric Renal Medicine
University of Bristol
Co-Founder of Purespring Therapeutics
What are the biggest challenges in the advanced therapy industry?
In my opinion, there are five challenges to overcome. The first is patient safety of new first-in-human therapies. The second is navigating the regulatory hurdles and approvals. The third is minimising drug dose and achieving local administration safely and efficiently. Another challenge is rolling out and testing individual therapies for small cohorts of patients for rare diseases. Finally, introducing novel vectors and delivery systems safely and fast enough is a big challenge.
What advanced therapy innovations are you most excited about?
I am excited about the ability to use gene editing tools and novel baculovirus delivery systems. It is exciting to see that more trials are happening, and reporting of these results (good and bad) takes the field forwards and identifies key hurdles. Finally, the manufacturing landscape is growing.
What are your predictions for the next 5 years for the Advanced Therapy Industry?
In renal medicine, we are progressing the field, with promising pre-clinical results and a concrete plan to achieve first-in-human therapy within a few years. More generally there will be broad adoption of AAV gene therapy for more and more diseases, with the issues of cost to health services needing to be solved at a national level. Furthermore, manufacturing will become more routine to access and commission.