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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
Targets and destroys repeat or non-repeat RNA (e.g. DM1, Huntington’s disease, SCA1, C9orf72-related ALS & FTD)

Destroy/Replace
Targets and destroys a toxic RNA while also replacing the gene (e.g. autosomal dominant retinitis pigmentosa)

Splicing
Targets and causes alternative splicing of mutation-carrying exons to restore functional protein products (e.g. Duchenne’s muscular dystrophy, Usher’s syndrome, frontotemporal dementia)

Translational Enhancement
Restores protein levels by increasing the function of a mutated gene (e.g. progranulin-related frontotemporal degeneration, retinitis pigmentosa, Dravet syndrome)

RNA Editing
Makes changes to RNA nucleotide sequences (e.g. point mutation genetic diseases)

SCIENTIFIC PUBLICATIONS AND POSTERS

SEPTEMBER 14, 2020, nature biomedical engineering
The Sustained Expression of Cas9 Targeting Toxic RNAs Reverses Disease Phenotypes in Mouse Models of Myotonic Dystrophy Type 1

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MAY 27, 2020
Locana Participates in the 25th Annual Meeting of the RNA Society to Share Research Regarding RNA-targeting Gene Therapy for Human Genetic Diseases
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MAY 13, 2020
Locana Presents Poster at ASGCT 2020: AAV9 Mediated Delivery of RNA Targeting CRISPR Corrects Molecular and Functional Phenotypes in Myotonic Dystrophy Type 1 Mouse Model
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AUGUST 2017, CELL
Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9
Batra, Nelles, Yeo, et al.
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