Another gene therapy company launches with VC cash and a CRISPR tech to target toxic RNA
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. That work was published in Cell.
Looking over their web site, the company plans to use a gene therapy vector to deliver an RNA-targeting protein with an enzyme at the tip.
The Locana platform Locana
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Rare genetic diseases and new gene therapy approaches have been all the rage now for a few years, coming after years of frustration when it comes to earlier attempts to address some of these diseases. So it’s no wonder that some high profile VCs are jumping into this for the ride.
Arch is leading the way on this new A round, which weighs in at $55 million — which looks like a tidy sum to get up to proof-of-concept work. Google’s GV is jumping in alongside UCB for the first time, joining Temasek and Lightstone Ventures — which have been involved from the start.
Locana is now a preclinical company, so getting into humans looks to be a top priority.
Along with the money, Locana is getting a CEO: Jeffrey Ostrove. Ostrove’s lengthy resume includes another CEO role at GenStem in San Diego, which is working on “lentiviral modified stem cell treatments for lysosomal storage and mitochondrial diseases.” He was also CEO of AbVitro, which Juno snapped up in a $125 million deal back in 2016.
David Nelles is a co-founder.