STAT+: Pioneering trial for treating genetic disease before birth nears reality

Summary: Dr. Tippi MacKenzie has spent 25 years trying to cure genetic diseases in babies before they are born. People used to tell her this amazing gene therapy was just "five years away." Now, it is finally happening. Her team asked the FDA to approve a trial for five unborn babies with a rare disease. The FDA even said they can skip animal testing because the treatment's tools are already proven safe.

For more details, see statnews at statnews.com/2026/05/20/tippi-mackenzie-in-utero-gene-therapy-nears-first-fda-trial/?utm_campaign=rss (opens in new tab)