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 Presents Preclinical Data from its Vectorized snRNA Exon Skipping Program for DMD and Cas13d Multi-targeting Program for C9orf72 ALS at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting
Locanabio Announces Equity Investment from CureDuchenne Ventures to Support Development of snRNA Exon Skipping Approach for Duchenne Muscular Dystrophy (DMD)