LEADING THE WAY IN
GENE THERAPIES TARGETING RNA
Locana is pioneering a new class of therapeutics by combining two validated approaches for treating diseases—gene therapy and RNA modification. The platform consists of several RNA-targeting systems that are combined with the power of gene therapy delivery to precisely modify dysfunctional RNA and create a novel class of genetic medicines to treat severe diseases.
LEADING THE WAY IN
GENE THERAPIES TARGETING RNA
Locana is pioneering a new class of therapeutics by combining two validated approaches for treating diseases—gene therapy and RNA modification. The platform consists of several RNA-targeting systems that are combined with the power of gene therapy delivery to precisely modify dysfunctional RNA and create a novel class of genetic medicines to treat severe diseases.

OUR PLATFORM TAKES A MULTI-FUNCTIONAL APPROACH TO MODIFYING DISEASE-CAUSING RNA

We are using gene therapy for delivering systems that are engineered to target and manipulate dysfunctional RNA involved in human disease. These systems can modulate RNA through different mechanisms, including destroying dysfunctional RNA, destroying dysfunctional RNA and replacing it with a normal gene, enhancing RNA translation to produce more of a specific protein, modifying splicing, and RNA editing.
Gene therapy
RNA modification systems are delivered to target tissue via an adeno-associated viral (AAV) vector

RNA TARGETING
RNA binding proteins and effector domains are designed to target disease-causing RNA

Multiple RNA
modification mechanisms
Our systems are designed to manipulate RNA in a variety of ways, providing a high degree optionality to address a broad spectrum of serious diseases

Destruction

Destroy/Replace

Splicing

Translational Enhancement

RNA Editing

SCIENTIFIC PUBLICATIONS AND POSTERS