Sustainable funding models for rare disease treatments, and in particular gene therapies, must be explored to make sure that patients can gain access to these therapies, according to Claire Booth, professor in gene therapy and pediatric immunology at the UK’s UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital (GOSH).
Not-For-Profit Models Needed For Commercially Unviable Rare Disease Drugs
Developing drugs for some rare diseases is “just not commercially feasible,” meaning that alternative approaches, such as funding through venture philanthropy and not-for-profit models, need to be explored, a gene therapy professor has said.
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