CNP saRNA

CNP saRNA is an experimental, preclinical self-amplifying RNA (saRNA) therapy designed for the treatment of achondroplasia. Developed by researchers at the University of Alberta, this therapeutic platform utilizes an alphavirus-derived synthetic RNA backbone to encode C-type natriuretic peptide (CNP). Unlike traditional recombinant protein therapies (such as vosoritide) that require daily injections, saRNA is designed to provide sustained, long-term expression of therapeutic proteins at lower doses by encoding its own replication machinery. The expressed CNP acts as an agonist on the natriuretic peptide receptor 2 (NPR2) in growth plate chondrocytes, stimulating endochondral bone growth and potentially offering a more cost-effective and accessible treatment option.

Other names

C-type natriuretic peptide self-amplifying RNACNP self-amplifying RNA