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

LEXINGTON, Mass. and AMSTERDAM, Nov. 03, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing ...

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

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...

A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...

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, ...

The goal of gene therapy is to permanently cure hereditary diseases. One of the most promising technologies for this is the ...

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 ...

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 ...

As third-quarter earnings continue to roll out, Novartis makes headlines with the second biggest acquisition of the year; ...