Biomodifying technologies Policy Briefing 1 “Making good targets for translational research”

Viable clinical applications for each of our case study technologies need to fit the available technological capacity for scale of production and delivery, to target a well-defined patient population where they can outcompete existing care regimens in cost-benefit assessment, and to align with regulatory requirements. We need to evaluate whether we can support multiple advanced therapies aiming at an initially limited set of conditions (for example both cell and gene therapies are in development for eye diseases like Advanced Macular Degeneration), and how we might do this. These initial product niches require support in ways that avoid ‘lock-in’ to one way of doing things, to enable the industry to evolve and expand over time.