Beyond innovation: why rare disease gene therapy needs a reset

Summary

This Expert Opinion article in Taylor & Francis looks at the challenges involved in developing gene therapies for rare diseases. The authors argue that scientific advances alone are not enough to bring these treatments to patients, and that more attention is needed to the wider process of developing and testing them.

The article considers issues including clinical trials, regulation and the development of gene therapies using technologies such as AAV and cell therapy. It calls for changes to help move promising treatments from research into clinical use more effectively.

Keywords: gene therapy, rare diseases, clinical trials, regulation, AAV, cell therapy, treatment development, translation