Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

Uppsala University Hospital-led investigators report that gene-edited donor islet cells survived 12 weeks inside a man with long-standing type 1 diabetes without any immunosuppressive medication.

Capsida Biotherapeutics has paused a gene therapy trial after the first patient died. The biotech, which began the phase 1/2 study in July, took the action to give it time to determine the “root cause ...