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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
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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