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.

Fractyl Health, Inc. has announced the submission of the first module of its Clinical Trial Application (CTA) for RJVA-001, a gene therapy candidate aimed at treating inadequately controlled type ...

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

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