It was time for Locana Inc., a San Diego-based biopharma, to grow itself, its science and its profile, according to newly installed director and CEO Jeffrey Ostrove, so the company sought out and just closed on an oversubscribed $55 million series A deal.

The funding is designed to expand Locana's RNA-targeting gene therapy programs and hone its platform. Ostrove is not disclosing which indications the company is considering or which will be its lead pipeline candidate, but propelling Locana into the clinic is the goal. For the moment, Locana, which focuses on genetic repeat expansion diseases using CRISPR gene editing techniques on RNA, has no set timelines.

"We'll advance some preclinical candidates toward the filing of an IND and even doing some clinical trials. Those are the high priorities," Ostrove told BioWorld.

The funding allows Locana to begin gathering human data and eventually move into the clinic. Ostrove said Locana has been approached to partner by a number of pharmas and large biotechs. The company will meet later this year or next with the most promising prospects to explore possibilities.

Ostrove has a long CV that includes co-founding and serving as CEO of Genstem Therapeutics, which worked on lentiviral modified stem treatments for lysosomal storage and mitochondrial diseases. He was also CEO of Abvitro Inc., which is now part of Celgene Corp. He has a PhD in molecular biology from the University of Florida and completed post-doctoral training at Johns Hopkins. He said he liked what he saw happening at Locana, adding that he would not have jumped into interacting with the company if he didn't feel good about the quality of the science.

"The science was incredibly powerful. It's a technology dealing with multiple diseases that have RNA in common," Ostrove said. "It's a very broad platform, meaning we have the ability to work on a large list of various types of diseases."

Huntington's disease and amyotrophic lateral sclerosis are often studied with CRISPR that targets RNA, but Ostrove would not commit to which diseases the company might investigate.

Also on the to-do list with the funding is expanding the management team and honing its platform, which was developed in the University of California, San Diego, in the laboratory of Eugene (Gene) Yeo, who also chairs Locana's scientific advisory board, and by Locana's chief technology officer and co-founder, David Nelles. Ron Batra, vice president of R&D, is a co-developer of the platform, which has been licensed from UC San Diego.

Fixing damaged RNA, as opposed to changing the DNA, is the process. The platform is constructed of modular components, including gene therapy vector, RNA-targeting protein and RNA-modifying enzymes, that support a range of therapeutic functions. It addresses DNA mutations through the RNA that produce them to eliminate off-target effect and potentially harmful changes in DNA.

Yeo and Nelles, along with four co-authors, put forth the initial platform approach three years ago, saying that RNA-programmed genome editing using CRISPR/Cas9 from Streptococcus pyogenes enables rapid and accessible alternation of specific genomic loci in many organisms. The goal is to track RNA in living cells in a programmable manner without genetically encoded tags, they wrote in their article, titled "Programmable RNA Tracking in Live Cells with CRISPR/Cas9," published in Cell magazine in April 2016.

The first round of seed funding came two years ago as did initiating scientific operations at JLABS in San Diego. Last year, Locana was selected for a nondilutive grant from the Muscular Dystrophy Association.

"People have been talking about gene therapy as medicine for the past 30 years, but the reality is that we're seeing some beautiful successes, whether they are with neurologic diseases or others," Ostrove said. "We believe our technology can create a paradigm shift."

Locana – the name is Sanskrit for illuminating or brightening – currently has about a dozen employees but the funding will change that. Ostrove said he will be adding about 20 to 25 people before the end of the year.

"It's important to hire people at all levels, lab workers and industry experts," he added.

The financing was led by Arch Venture Partners along with Temasek Holdings and Lightstone Ventures, which are existing investors. The new investors include UCB Ventures and GV (formerly Google Ventures).

The company directors include Chairman Tom Daniel, of Arch Venture Partners; Cayce Denton, of Temasek; Jean George, of Lightstone Ventures; plus and Ostrove and Yeo. Emmanuel Lacroix is an observer for UCB Ventures.

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