In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns and their families. Now, we’re taking an even earlier step: treating ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages.

RNA gene scissors (CRISPR-Cas13) are gaining significant attention as a next-generation gene therapy with fewer side effects.

The company is one of the first to receive a “platform technology designation,” which could speed the review of certain gene ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...