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

DFS at 24 months improved from 79% with observation to 96% with the use of a 14-gene assay to guide decision making about ...

This first-in-human trial is testing a highly unconventional method: delivering fat-derived stem cells from the patient ...

Greater numbers of circulating stem cells increase the efficacy of in vivo gene therapy techniques, potentially reducing the price tag of lifesaving treatments.

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

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

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

An international group of gene editing leaders has put out a call for a 10-year ban on heritable human genome editing (HHGE), ...

While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of ...

A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a ...

Rocket Pharmaceuticals Inc (NASDAQ:RCKT) on Tuesday announced an update related to RP-A501, its investigational gene therapy ...

Find out if gene therapy has also find its way into skin diseases. With two approvals in the last 2 years, is it the start of ...