In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

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

Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

Machine learning models have seeped into the fabric of our lives, from curating playlists to explaining hard concepts in a ...

KJ Muldoon's life-threatening disorder, called severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, was treated with ...

KJ Muldoon, a 10-month-old baby diagnosed with a rare metabolic disease called CPS1 has been at Children Hospital of ...

Scientists at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, have found that gene editing using ...

This study reveals the potential of engineered phage-IL-12 therapy in melanoma, enhancing immune response and tumor ...

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

The U.S. FDA has paused Rocket Pharmaceuticals' mid-stage gene therapy trial after a patient died from serious complications, ...

A 5-year-old boy from Center City has faced rare odds since birth and now this warrior is in a real life race to rewrite his story.

The FDA has slapped a clinical hold on Rocket Pharmaceuticals’ pivotal gene therapy trial in response to a death. Rocket, ...