Gene therapy coming<br>of age: Opportunities<br>and challenges to<br>getting ahead

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 RNAand 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.