Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Ultragenyx Pharmaceutical (RARE) announced on Thursday that its gene therapy candidate DTX301 reached the main goal in a late-stage trial for ornithine transcarbamylase (OTC) deficiency, the most ...

Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the burden of lifelong transfusions. A single one-time gene therapy could free ...

Explore the future of gene therapy delivery with lipid nanoparticles. Learn how they can transform treatments for various diseases.

A new chapter is unfolding for gene therapies in Europe. With retinal programs advancing into Phase I/II trials and regulators introducing more flexible approval pathways for rare diseases, the field ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

Since scientists first discovered that human immune cells could be modified to become cancer-fighting agents, they've been trying to engineer a cell that's effective against solid tumors, which ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.