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Locanabio’s Proprietary CORRECTx™ RNA-Targeting Gene Therapy Reduces Disease-Causing Hexanucleotide Repeat Expansions in a Model of Amyotrophic Lateral Sclerosis

Oral presentation of new preclinical data at American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting –

Excerpt from the Press Release:

SAN DIEGO, May 19, 2022 /PRNewswire/ — Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with severe neuromuscular and neurodegenerative diseases, today announced the presentation of new preclinical data on its proprietary CORRECTx™ platform for the potential treatment of amyotrophic lateral sclerosis (ALS). The data, generated in vitro and in a mouse model of ALS, demonstrate that a single administration of the CORRECTx RNA-targeting gene therapy reduced toxic RNA transcripts from hexanucleotide (G4C2) repeat expansions in the chromosome 9 open reading frame 72 (C9ORF72) gene that are known to be associated with ALS in humans.

“These new data demonstrate the potential advantages of our CORRECTx technology, which can be designed to selectively target both disease-causing sense and antisense RNA transcripts in a single construct while sparing expression of the wild-type C9ORF72 allele which may be important for cellular function,” said John Leonard, Ph.D., chief scientific officer of Locanabio. “These studies support further development of our CRISPR-Cas13d multi-guide RNA-targeting system as a potential therapeutic approach for ALS and other neurologic disorders associated with this locus.”

A G4C2 hexanucleotide repeat expansion in the C9ORF72 gene is the most common genetic cause of familial ALS and frontotemporal dementia (FTD). Transcription at the C9ORF72 repeat locus produces both sense (GGGGCC or G4C2) and antisense (CCCCGG or C4G2) RNA transcripts that accumulate as foci in the nucleus as well as dipeptide repeat protein products that aggregate and cause neuronal toxicity in the brain and spinal cord. In the studies presented at ASGCT, Locanabio used its CORRECTx platform comprising a CRISPR-Cas13d system with two guide RNAs designed to target both disease-causing sense and antisense RNA repeats packaged into a single AAV9 vector for delivery. Significant reduction of toxic RNA products was observed in isolated cells. Following one-time administration of the RNA-targeting CRISPR-Cas13d system in the C9ORF72 ALS model, a decrease in toxic G4C2 transcripts was observed without reduction in total C9ORF72 RNA levels. Additional data demonstrate that the expression of the CRISPR-Cas13d multi-guide system was well tolerated post-administration throughout the 10-week study.

“The versatility of our selective CORRECTx RNA-targeting approach provides Locanabio with several advantages as we advance development candidates from our novel class of genetic medicines to other disease targets,” said James Burns, Ph.D., chief executive officer of Locanabio. “We are pleased with the progress of our ALS program and are very encouraged by these data presented today, which supports our plans to leverage the multi-targeting and mutation specific capabilities of the CORRECTx platform to expand our pipeline and treat other serious diseases with limited or no treatment options.”

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