As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

AMSTERDAM, May 29, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the ...

Wafik El-Deiry, a Brown University cancer researcher and pandemic-era free speech advocate, is calling for a budget for the ...

The biotech plans to launch other trials for NRTX-1001 in neocortical focal epilepsy and other areas ... shifts away from the entire cell and gene therapy field, layoffs have hit the landscape ...

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...

In a stunning medical breakthrough that effectively brings designer babies out of the realm of science fiction, a ...

“People who take drugs for epilepsy often complain that the ... The Allen Institute in Seattle have created a new gene replacement therapy in mice that could lead to better treatments in humans.

Researchers have developed a new suite of genetic tools for cell-type specific targeting to battle brain disease.

Scientists say they've put together a molecular toolkit that could be used to treat a variety of brain diseases, possibly ...