Rare Diseases – Clinical & Commercial Developments
Guest Editors: Dr Sven Kili, GSK & Dr Jaap Boelens, UMC Utrecht
The promising early clinical data from gene therapies for rare diseases has seen a resurgence in investment and optimism in the field. This spotlight will discuss the current clinical landscape as well as the critical considerations in moving these therapies towards successful commercialization:
- Navigating the transition from academia to early proof-of-concept studies and onto commercialization, including the selection of appropriate clinical end points.
- The latest clinical developments and outcomes of gene therapies for rare diseases of the CNS, metabolism, eye and liver.
- What can we learn from the commercial performance of these therapies thus far?
- What should the reimbursement price point be and how can companies establish a viable business strategy to support different payment models?