Rare-disease gene therapy after the first wave
After the first wave of approvals, the durability and access questions around rare-disease gene therapy are coming into focus.
The first wave of rare-disease gene therapies established that the science works. The durability story, the cost-of-goods reality, and the access economics are still being written - and that is where the next chapter is decided.
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Published by PatientSpotlight, by PanaceaIntel.