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

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

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

The U.S. FDA has paused a mid-stage gene therapy trial by Rocket Pharmaceuticals after a patient suffered serious ...

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

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

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...

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

Intellia Therapeutics’ stock may have taken a hit in the wake of details of a grade 4 adverse event in a phase 3 trial, but ...

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

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