A large dog showing affection to a young woman in the woods

Locanabio Announces Upcoming Preclinical Data Presentations at American Society of Gene and Cell Therapy Annual Meeting

SAN DIEGO, May 5, 2021 – Locanabio, Inc., an RNA-targeting gene therapy company focused on developing life-changing therapies for patients with severe neurodegenerative, neuromuscular and retinal diseases, today announced that new data from its preclinical research will be highlighted in oral and poster presentations at the 24th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting, taking place virtually from May 11-14, 2021. The presentations include results from a preclinical in vivo study showing a novel PUF RNA-binding protein system delivered via an adeno-associated virus serotype 9 (AAV9) vector is safe and effective in eliminating toxic CUG repeats in a mouse model of myotonic dystrophy type 1 (DM1). Data also provide insights on the potential of a unique CRISPR/Cas13d gene therapy system for skipping exon 13 mutations in the USH2A gene that are associated with usher syndrome type 2 and non-syndromic autosomal recessive retinitis pigmentosa.

Details of the presentations at ASGCT are as follows:

Oral Presentation
Title: AAV9 Mediated Delivery of PUF RNA Targeting System Corrects Molecular and Functional Defects in a Myotonic Dystrophy Type 1 Mouse Model
Session Title: AAV Therapies for Neurological and Sensory Diseases
Authors: Ranjan Batra, Daniela Roth, Haydee Gutierrez, Nandini Narayan, Greg Nachtrab, Claire Geddes, Aaron Berlin, and Dan Gibbs
Presentation Date and Time: Thursday, May 13, 2021 at 5:45 – 6:00 p.m. EDT

Poster Presentations
Title: Novel RNA-Targeting Gene Therapy Approach for Usher’s Syndrome Type II Retinitis Pigmentosa (Poster ID #100)
Session Title: Neurological Diseases
Authors: Dan Gibbs, Rea Lardelli, Greg Nachtrab, Daniela Martino Roth, Shawn Lee, Claire Geddes, Alistair Wilson, Nandini Narayan, Dimitrios Zisoulis, and Ranjan Batra
Presentation Date and Time: Tuesday, May 11, 2021 at 8:00 a.m. EDT

Title: Rational Design of Short Constitutive Promoters for Improved Transgene Expression in AAV Vectors (Poster ID #281)
Session Title: AAV Vectors – Virology and Vectorology
Authors: Greg Nachtrab, Scott Davis, Daniela Martino Roth, Dimitrios Angelis, Alistair Wilson, Claire Geddes, Nandini Narayan, Ranjan Batra, and Dan Gibbs
Presentation Date and Time: Tuesday, May 11, 2021 at 8:00 a.m. EDT
The study abstracts are available on the ASGCT meeting website and can be accessed via the following link:

About Locanabio, Inc.

Locanabio is the global leader in developing a new class of genetic medicines. Our unique and multidimensional approach uses gene therapy to deliver RNA binding protein-based systems to correct the message of disease-causing RNA and thereby change the lives of patients with devastating genetic diseases. These broad capabilities delivered via gene therapy enable
Locanabio to potentially address a wide range of severe diseases with a single administration. The company is currently advancing programs in neurodegenerative, neuromuscular and retinal diseases. For more information, visit

Media Contact

Jenna Urban
Berry & Company