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.
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.
Locanabio is a leader in developing a new class of genetic medicines that has the potential to significantly improve the lives of patients with devastating genetic diseases by correcting the message of disease-causing RNA. Our proprietary platform, CORRECTx™, uses gene therapy to deliver RNA-binding protein systems that can be engineered to selectively manipulate disease-causing RNA by multiple mechanisms.
Our systems are designed to provide a durable therapy with a single administration without altering a cell’s DNA. Locanabio’s CORRECTx platform has applications across a range of tissues and diseases and we are currently advancing programs in neurodegenerative and neuromuscular diseases. For more information, visit www.locanabio.com
This article was shared with Prittle Prattle News as a Press Release.
