Myrtelle Announces Nature Medicine Publication of Interim Results from Its Phase 1/2 Clinical Trial of Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease

Groundbreaking study demonstrates that targeted oligodendrocyte gene therapy leads to decreased N-acetylaspartate (NAA) levels and increased ...
Ars Technica

From birth to gene-edited in 6 months: Custom therapy breaks speed limits

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects an ...
BioPharma Dive

UniQure to seek approval of Huntington’s gene therapy after trial win

New study data showed treatment slowed signs of disease progression by 75% after three years, a finding that could have ...

Preliminary but promising gene therapy shows 75% reduction in Huntington's disease progression

A one-time experimental treatment shows unprecedented success in slowing Huntington's disease, according to UCL researchers. FDA review expected in 2026.