SAN DIEGO, May 2, 2022 /PRNewswire/ — Locanabio, Inc., a genetic drug company developing RNA-targeted therapies for patients with severe neuromuscular and neurodegenerative diseases, today announced that new data from preclinical research of its platform proprietary CORRECTx™ in C9ORF72-mediated amyotrophic lateral sclerosis (ALS) will be highlighted in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 25and Annual meeting, which takes place May 16-19, 2022 in washington d.c.
The details of the oral presentation at the ASGCT are as follows:
Title: AAV9 Delivery of RNA Targeting Systems Eliminates Hexanucleotide Repeat Expansions in C9ORF72 ALS/FTD Models
Session title: Breakthroughs in neuromuscular and auditory disorders
Speaker: Jeannie ChewPh.D., Locanabio
Presentation date and time: Thursday, May 19, 2022 from 10:45 a.m. – 11:00 a.m. ET
Abstract number: 1224
The summary of the study is available on the ASGCT meeting website and can be accessed via the following link: https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=2119
Repeated hexanucleotide expansions in the C9ORF72 gene are the most common cause of familial ALS, leading to the accumulation of toxic RNA foci, including sense RNA, G4C2, and antisense RNA, C4G2. These RNA foci form secondary RNA structures that sequester regulatory RNA-binding proteins, causing dysregulation of RNA splicing and trafficking. Translation of sense and antisense RNA strands also forms potentially toxic repeating dipeptide proteins. Leveraging the CORRECTx platform, Locanabio is able to design multi-targeting constructs, delivered by gene therapy, that can reduce both pathogenic sense and antisense RNA transcripts and maintain allelic selectivity to preserve the wild-type C9ORF72 allele in ALS patients.
About Locanabio, Inc.
Locanabio is a leader in the development of a new class of genetic drugs that has the potential to dramatically improve the lives of patients with devastating genetic diseases by correcting the message of pathogenic RNA. Our proprietary platform, CORRECTx™, uses gene therapy to provide RNA-binding protein systems that can be engineered to selectively manipulate pathogenic RNA through multiple mechanisms. Our systems are designed to provide long-lasting therapy with a single administration without altering a cell’s DNA. Locanabio’s CORRECTx platform has applications in a range of tissues and diseases and we are currently advancing programs in neurodegenerative and neuromuscular diseases. For more information, visit www.locanabio.com.
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SOURCE Locanabio, Inc.