Locanabio, Inc. today announced that new data from preclinical research of its proprietary CORRECTx™ platform in C9ORF72-mediated amyotrophic lateral sclerosis (ALS) will be highlighted in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting.
| SAN DIEGO, May 2, 2022 /PRNewswire/ -- Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with severe neuromuscular and neurodegenerative diseases, today announced that new data from preclinical research of its proprietary CORRECTx™ platform in C9ORF72-mediated amyotrophic lateral sclerosis (ALS) will be highlighted in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting, taking place May 16-19, 2022 in Washington, D.C. Details of the oral presentation at ASGCT are as follows: Title: AAV9-Mediated Delivery of RNA Targeting Systems Eliminates Hexanucleotide Repeat Expansions in C9ORF72 ALS/FTD Models The study abstract is available on the ASGCT meeting website and can be accessed via the following link: https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=2119 Hexanucleotide repeat expansions in the C9ORF72 gene are the most common cause of familial ALS, leading to the accumulation of toxic RNA foci, including both sense RNA, G4C2, and antisense RNA, C4G2. These RNA foci form secondary RNA structures that sequester regulatory RNA-binding proteins, which causes dysregulation of RNA splicing and trafficking. The translation of the sense and antisense RNA strands also forms potentially toxic dipeptide repeat proteins. Leveraging the CORRECTx platform, Locanabio is able to design multi-targeting constructs, delivered via gene therapy, that can reduce both disease-causing sense and antisense RNA transcripts and maintain allele selectivity to preserve the wild-type C9ORF72 allele in patients with ALS. About Locanabio, Inc. Media Contact SOURCE Locanabio, Inc. |
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