C9ORF72-ALS therapeutic
/ Locana
- LARVOL DELTA
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May 16, 2023
Locanabio Presents Preclinical Data from its Vectorized snRNA Exon Skipping Program for DMD and Cas13d Multi-targeting Program for C9orf72 ALS at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting
(BioSpace)
- "Locanabio...announced the presentation of data from its lead vectorized snRNA exon skipping program, LBIO-115, in development for the treatment of Duchenne muscular dystrophy (DMD). A second presentation highlights data from the company’s Cas13d multi-targeting program for C9orf72-related amyotrophic lateral sclerosis (ALS). The ASGCT 26th Annual Meeting is being held May 16-20, in Los Angeles. 'The preclinical data presented at the ASGCT meeting highlight Locanabio’s expertise in therapeutic RNA modification with two different payloads from our versatile drug development platform: our proprietary vectorized snRNAs and Cas13d which are designed to durably address disease causing RNA using different mechanisms.'"
Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders
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