UCSD-Derived  Gene Therapy Technology Secures $55 Million

May 26, 2019 | CRISPR, Genetics, RNA


Bradley J. Fikes of the San Diego Union Tribune reports  San Diego-based Locana has raised $55 million in venture capital funds. The biotech startup targets abnormal RNA instead of DNA to treat genetic diseases.


Locana’s gene therapy technology was developed within University of California, San Diego. Privately held, Locana focuses on the creation of cures to diseases that are caused by abnormally repeated genetic sequences. Examples of such diseases includes Huntington’s disease and forms of myotonic dystrophy.

The Technology

Locana’s underlying technology, a CRISPR DNA editing system, was adapted by UCSD’s Gene Yeo and David Nelles, to target RNA, the messenger molecule made from DNA.  In 2017 they reported they would commercialize the technology. Both individual’s co-founded the company in 2016.  The two envisioned a CRISPR system that could be carried into cells by an adeno-associated virus, a common delivery vehicle—like a highly for genes into cells.

They are creating a new class of precision medicines to treat a range of human genetic diseases. Their RNA-targeting technology platform affords them to precisely address these mutations on the level of RNA. By targeting RNA, their approach avoids the risk of off-target effects in DNA and is suited to address many diseases linked to dysfunctional processing of RNA.

The wide range of potential indications is supported by the modularity of their RNA-targeting gene therapies which are composed of interchangeable components that provide considerable control of specificity and activity.

They believe they are in a leadership position when it comes to RNA biology and RNA-mediated disease.

The People

  • Gene Yeo, Founder
  • David Nelles, Founder

The Investors

The Series A financing was led by existing investors ARCH Venture Partners with Temasek and Lightstone Ventures. New investors include UCB Ventures and GV (formerly Google Ventures).


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