Opus Genetics (IRD) announced that its work was featured on Good Morning America in honor of World Blindness Awareness Month. The feature ...

The national feature highlighted the story of Lindsey Rambo, the second participant in Opus Genetics’ ongoing Phase 1/2 trial of gene therapy OPGx-LCA5, aimed at restoring vision for people living ...

The novel treatment, partly developed by CHOP, eliminates the need for regular infusions while preventing the ...

In a world first, a bespoke gene-editing therapy benefitted one child. Now reseachers plan to launch a clinical trial of the ...

Children with hereditary deafness regained their hearing thanks to a type of gene therapy, a new study published on Wednesday found. In a clinical trial, co-led by investigators from Mass Eye and Ear, ...

Scientists at The University of Texas at Austin have developed a revolutionary gene-editing method using bacterial retrons ...

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...

Researchers in the United Kingdom say they have successfully trialed what could become the world's first gene therapy for Huntington's disease – a fatal neurodegenerative disorder that is typically ...

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.

An increasing demand for gene therapies is focusing the industry on enhancing production capabilities, transitioning from ...

Lilly’s upfront offer for Adverum, the developer of a gene therapy for age-related macular degeneration, is less than the ...