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

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 ...

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

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

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

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

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 ...

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

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment May 15, 2025 — A research team has developed and safely delivered a personalized gene ...

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 ...