Many rare diseases are caused by genetic mutations resulting in disease-causing RNA transcripts. Locanabio’s pioneering platform of RNA-binding systems specifically targets these RNA transcripts and modifies them.
The versatility of our RNA-binding systems allows us to use a variety of mechanisms to target and correct faulty RNA. By combining the specificity of RNA-binding systems with the lasting effect of a one-time gene therapy, we can durably address the disease-causing RNA in patients suffering from a range of rare genetic neuromuscular and neurodegenerative diseases without altering their DNA.
Locanabio Announces Presentation of Preclinical Data Demonstrating an Improved Vectorized snRNA Platform with Applications in Exon Skipping for the Potential Treatment of Duchenne Muscular Dystrophy (DMD) at the 28th Annual Congress of the World Muscle Society (WMS2023)