Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...

The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...

The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...

A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.

ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...

Lilly and German biotech Seamless Therapeutics will develop recombinase-based treatments for hearing loss.

Ultragenyx now expects a 6-month review period for the therapy and a likely decision from the FDA regarding potential ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

Future GLP-1 gene therapy could enable long-term hormone production, with Fractyl Health and others planning human trials in 2024.

Studies of Hunter and Hurler syndromes on hold ...

Hydrocephalus is a life-threatening condition that occurs in about 1 in 1,000 newborns and is often treated with invasive ...

After finding an intraventricular tumor in one clinical trial participant, the FDA placed a clinical hold on an experimental ...