LEADING THE WAY IN
RNA TARGETED GENE THERAPIES
Locanabio is creating a new class of genetic medicines that combines the two leading approaches for treating genetic diseases —RNA modification and gene therapy. Locanabio’s new approach to RNA modification leverages our proprietary platform of RNA-binding protein systems. These versatile systems are designed to precisely correct dysfunctional RNA in a variety of ways. The genetic instructions to produce these systems are packaged into a single adeno-associated virus (AAV) vector and administered as a one-time treatment.
LEADING THE WAY IN
RNA TARGETED GENE THERAPIES
Locanabio is creating a new class of genetic medicines that combines the two leading approaches for treating genetic diseases —RNA modification and gene therapy. Locanabio’s new approach to RNA modification leverages our proprietary platform of RNA-binding protein systems. These versatile systems are designed to precisely correct dysfunctional RNA in a variety of ways. The genetic instructions to produce these systems are packaged into a single adeno-associated virus (AAV) vector and administered as a one-time treatment.

A MULTI-FUNCTIONAL APPROACH TO MODIFYING DISEASE-CAUSING RNA

We are building a broad pipeline of RNA-modifying gene therapies. Our platform allows for the development of modular systems that can modify RNA in a variety of ways: specifically destroy toxic RNA, correct splicing by excluding mutated exons, destroy faulty RNA and replace it with a functional gene, enhance translation to express more protein and correct point mutations with RNA editing. Our unique approach uses the appropriate RNA modification technique to target a specific disease.
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