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