As Eli Lilly awaits an FDA approval decision for its oral GLP-1 orforglipron, the pharma has discarded three clinical-stage ...

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus. The ...

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 ...

Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

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 ...

In late January and early February 2026, Ultragenyx Pharmaceutical announced positive long-term clinical data for UX111, its ...

Two-year follow-up data demonstrate sustained benefit, including improvements in pain, fatigue and bone health, following single low dose of ...

A central nervous system (CNS) tumor has prompted the FDA to place clinical holds on two Regenxbio gene therapies, including ...

There are currently no approved disease-modifying therapies for MPS IIIA.

Treatment with onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA) type 1 was associated with less need for nutritional support or nighttime ventilation compared to recommended ...

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.