The completion of the first draft of the human genome in 2001 was supposed to kick off an era of personalized medicine and curative gene therapies.1 Only in the past few years has that promise started to become reality: several RNA- and DNA-based therapies are now on market, and the first curative gene therapy, Luxturna, was approved in 2018.
These successes were largely due to a better clinical and scientific understanding of safety profiles as well as a refined manufacturing process that met the consistency and quality standards required for clinical scale. The bevy of new gene therapies in the development pipeline has the potential to transform care across several therapeutic areas. However, it also creates new challenges for key stakeholders—including pharma companies, regulatory agencies, providers and payers—in how to recalibrate the pharma development and reimbursement model for therapies that go beyond our traditional approach to treating disease.
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By Emily Capra, Jeff Smith, and Guang Yang
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