The company intends to divest Roctavian and “remove it from our portfolio,” CEO Alexander Hardy said, after nearly three ...

However, in late September 2025, scientists successfully treated Huntington’s for the first time in a ground-breaking study.

In the small first-in-human study, AB-1002 showed an encouraging safety profile and early signs of efficacy, investigators ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Viral vectors dominate gene therapy, with lentivirus, adenovirus, and AAV being key players, each with unique advantages and limitations. Non-viral vectors, such as lipid nanoparticles and GalNAc, ...

Researchers have unveiled a new DNA-based therapy that silences the PCSK9 gene to lower cholesterol without statin side effects. Early results show dramatic reductions in cholesterol levels, offering ...

Researchers in the lab of Hans-Peter Kiem, MD, PhD, at Fred Hutch Cancer Center have devised a method that could one day treat genetic hematologic disorders by correcting how the body makes blood ...

Research Ireland and APC will together fund new research into how advanced gene therapies are created and delivered.

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...