RNA-targeting gene therapy
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Locanabio is developing gene therapies that correct RNA molecules. DNA mutations in gene coding sequences that cause disease are also present in the messenger RNA (mRNA) intermediate. While many current gene therapies target DNA, Locanabio is targeting RNA mutations. Locana’s CORRECTX platform enables screening of RNA binding proteins that precisely target and correct dysfunctional mRNA that causes many genetic diseases. Targeted RNA can be corrected through cleavage, splicing gene replacement, translation enhancement and editing, all of which Locanabio is investigating. Locanabio’s pipeline is in preclinical stages and focuses on therapies for neurodegenerative, neuromuscular and retinal diseases.