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

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 ...

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

Dr. Tejal Aslesh Tailor is an accomplished neuroscientist and molecular biologist with years of expertise in preclinical ...

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

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 ...

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 ...

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

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