A gene therapy trial claims to show promising results for patients with Huntington’s disease. A press announcement of the experimental treatment suggests significant slowing of the disease over three ...

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

Opus Genetics today announced the positive 3-month pediatric data from its Phase 1/2 open-label, ascending-dose study of the ...

As well as slowing disease progression by 75 percent at 36 months, the scientists found that neurofilament light protein (NfL)—a biomarker of neuronal damage—levels in spinal fluid were lower in ...

September is Sickle Cell Awareness Month, and millions of people worldwide are living with the disease. But advances in ...

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

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

The new therapy is being hailed as a breakthrough. But the small study has some limitations – and hasn’t been peer-reviewed.

Cancer scientists have been working against the clock for decades to outsmart a disease that comes back more powerfully after ...

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

UC Davis Health researchers are testing a new gene therapy that could be transformational for patients with Angelman syndrome ...

Ten-year-old Sebastian Veiman is racing against time to receive €2.5 million gene therapy that could halt — or even reverse — ...