article thumbnail

Rare diseases, repurposing and the role of AI

pharmaphorum

These might be licensed drugs that could hold potential for a patent extension, or drugs which failed efficacy trials for an intended indication. Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise.

article thumbnail

What challenges does the advanced therapies sector face? 

Drug Discovery World

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Intellectual property: High patent royalties and licensing fees for key technologies can inflate treatment costs. Fostering open innovation and collaboration could help address this issue.