Mei’s Story: Investigation Reveals Unreported Fatal Outcome After In Vivo Base Editing of CHD3
In March 2025, a six-year-old girl received the first reported in vivo base-editing treatment directed at the human CNS. The girl, given the pseudonym “Mei” — meaning “beautiful” in Chinese — had Snijders Blok-Campeau syndrome, an extremely rare genetic condition associated with mutations in ...
Roche Ends Two HD Clinical Trials as Other Huntingtin-Lowering Therapies Advance
On July 9, Roche announced it would discontinue two ongoing clinical studies investigating treatments for Huntington's disease (HD) after separate analyses showed the programs would not achieve their intended goals. While the news is a significant disappointment for the HD community, both studies have ...
