RNA-targeting gene therapy

A flexible and specific way to address human disease

Why target RNA?

Differences in our DNA have consequences ranging from inability to enjoy cilantro to devastating genetic disease. But one thing is the same among many of these mutated DNA sequences — targeting the RNA produced by them is a powerful way to treat them.


One reason to address DNA mutations through the RNA they produce is to eliminate off-target effects (potentially harmful changes) in DNA. 

Another reason is linked to the biology of specific diseases. Many are caused by dysfunctional RNA behavior — conditions our platform is well-suited to address.

For these reasons and more, we treat disease by targeting RNA.

Our platform is composed of modular components, supporting a wide range of therapeutic functions and specificities.

RNA-targeting protein

Gene therapy vector

RNA-modifying enzyme


The Locana  Platform

Our RNA-targeting gene therapy platform is a powerful way to address the root cause of genetic disease. 



RNA processing

Adeno-associated viral particles

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Tissue-targeted delivery 


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April 2016, Cell

Programmable RNA Tracking in Live Cells with CRISPR/Cas9

Nelles, Yeo, et al.

August 2017, Cell

Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9

Batra, Nelles, Yeo, et al.

Locana means 'illuminating' or 'brightening' in Sanskrit.

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