Locanabio to report preclinical data from snRNA exon skipping program in DMD and Cas13d-mediated C9orf72 ALS program
SAN DIEGO, May 02, 2023 (GLOBE NEWSWIRE) -- Locanabio, Inc., a genetic medicines company developing RNA-targeted therapeutics for patients with rare genetic neuromuscular and neurodegenerative diseases, today announced that data from LBIO-115, its vectorized snRNA exon skipping program in development for the treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, and its Cas13d-mediated program for C9orf72-related amyotrophic lateral sclerosis (ALS) will be presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting. The ASGCT meeting is being held from May 16-20, 2023, in Los Angeles, CA.
Details of Locanabio’s ASGCT Presentation Abstracts
Title: Small Nuclear RNA-mediated Exon51 Skipping AAV9 Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
Presenting Author: Rea Lardelli Markmiller, Ph.D.
Session Title: Thursday Poster Session
Session Date and Time: Thursday May 18, 2023, Noon – 2:00 pm PT
Abstract Number: 1198
Title: Cas13d Multi-targeting Efficiently Targets Sense and Antisense HRE Containing Toxic RNAs and poly-GP DPR in C9ALS Patient Cells and in C9-BAC500 Mouse Model
Presenting Author: Daniela Martino Roth, Ph.D.
Session Title: Thursday Poster Session
Session Date and Time: Thursday May 18, 2023, Noon – 2:00 pm PT
Abstract Number: 811
All posters will be available for registered attendees for on-demand viewing on the ASGCT website on May 16, 2023, beginning at 6:00 AM ET. Upon release at ASGCT, Locanabio’s presentations will also be available on the scientific resources page of Locanabio’s website.
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 uses gene therapy to deliver RNA-binding systems, including snRNA, Cas13d, and PUF 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 platform has applications across a range of tissues and diseases, and we are currently advancing programs in rare genetic neuromuscular and neurodegenerative diseases. For more information, visit www.locanabio.com.
Investor and Media Contacts:
Sylvia Wheeler
Wheelhouse LSA
swheeler@wheelhouselsa.com
Elizabeth Wolffe, Ph.D.
Wheelhouse LSA
lwolffe@wheelhouselsa.com
