Locana bags $55M for RNA-targeting gene therapies

Locana CEO Jeffrey Ostrove, Ph.D. (Locana)

Locana, a biotech working on gene therapies that target RNA rather than DNA, raised $55 million to expand its platform and pipeline as well as to build out its leadership team. The company is taking aim at diseases such as Huntington’s and amyotrophic lateral sclerosis (ALS) that are caused by problems in RNA that stop cells from producing essential proteins. 

The financing comes from Locana’s backers ARCH Venture Partners, Temasek and Lightstone Ventures along with a pair of new investors: GV and UCB Ventures.

Locana's work is based on technology from the lab of Gene Yeo, Ph.D., a professor of cellular and molecular medicine at the University of California, San Diego. The company's chief technology officer, David Nelles, Ph.D., and R&D chief Ranjan Batra, Ph.D., also worked on the platform in Yeo’s lab.  

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Locana is keeping mum on the diseases it’s interested in, saying only that it's done preclinical work in multiple microsatellite repeat expansion diseases. 

“There is a laundry list of dozens and dozens of diseases that have repeat elements in them,” Locana CEO Jeffrey Ostrove, Ph.D., told FierceBiotech. In these diseases, too many repeats of RNA sequences causes RNA to clump together in cells, preventing them from making proteins. In 2017, Nelles and Batra authored a study showing their RNA-targeting Cas9 system cleared 95% of RNA clumps in cells taken from patients with Huntington’s disease and myotonic dystrophy, a type of ALS. 

RELATED: RNA-targeting CRISPR could yield treatments for Huntington's, ALS 

Making changes to RNA rather than DNA means any gene editing will not be permanent. What’s more, Ostrove said, it can address safety concerns of editing DNA such as off-target mutations, where gene-editing systems make unintended edits that lead to harmful side effects. 

Some companies are working to drug RNA, using proteins to modify or destroy RNA and change the biology within a cell at the RNA level, Ostrove said. Other companies are taking a different tack, such as targeting messenger RNA with antisense molecules. What sets Locana apart is the one-time aspect of using gene therapy rather than a drug that, presumably, must be given over and over again, he said. 

“We are using an automated system we developed to look at literally thousands of different combinations of RNA binding-proteins and effector proteins to study many different diseases. It really opens Locana up to a lot of partnering opportunities,” Ostrove said. 

Some biotech and pharma suitors have already approached the company, but Locana is focusing in the near term on fine-tuning its technology and bolstering its management and scientific teams. The company expects to add between 20 to 25 staffers by the end of the year.

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