Huntington’s disease has been successfully treated for the first time using a gene therapy, which may be available in the US as soon as next year ...

A company called uniQure has announced promising results from a trial of a new gene therapy for Huntington's disease. The ...

A one-time experimental treatment shows unprecedented success in slowing Huntington's disease, according to UCL researchers.

The AAV pullback comes amid Biogen’s aggressive cost-cutting campaign, which put some 1,000 jobs on the chopping block with ...

Scientists from Tel Aviv University and Boston Children's Hospital use a optimized virus to deliver a corrected gene into the ...

Huntington’s is caused by a single error in our DNA, a “typo” in a gene responsible for creating a protein called huntingtin.

Stanford researchers and their collaborators have revealed a new device that could change the way scientists conduct ...

After three patients died, the maker of an investigational gene therapy stopped shipments of the drug after it refused to ...

Jesse Gelsinger died after receiving a gene therapy treatment to treat a liver disease. The death sparked an investigation ...

At the European Cell & Gene Therapy Summit 2025, funding challenges and innovation drove discussions on the sector’s future.

"On the basis of these results, it seems likely AMT-130 will be the first licensed treatment to slow Huntington's disease, ...

The gene therapy company uniQure has announced top-level data from its trial of AMT-130, a new gene therapy designed to treat ...