A San Diego-based biotech has attracted substantial venture support for its clinical foray into gene therapies that can target toxic brands of RNA ? sparked by DNA mutations ? that trigger genetic diseases. And they?ve wooed a well-known West Coast entrepreneur to the helm.
The biotech is called Locana, which is working on the scientific explorations done by UC San Diego professor Gene Yeo. Yeo?s published work includes lab experiments that used CRISPR Cas9 tech to modify repetitive RNA ?an approach with implications for some tough diseases like myotonic dystrophy type?1 and 2, Huntington?s disease, and C9orf72-linked amyotrophic lateral sclerosis.