Harnessing mRNA-display for the Discovery of Macrocyclic Peptide Drugs
Robert Hammer
Senior Principal Scientist & Group Leader, Ra Pharmaceuticals
Ra Pharmaceuticals is developing a new class of drugs with the diversity and specificity of antibodies, coupled with the bioavailability of small molecules. Our macrocyclic peptide-like molecules are highly-stable, synthetic products suitable for targeting both extracellular and intracellular proteins. Ra uses in vitro mRNA display technology to create libraries of up to 100 trillion drug-like molecules and directly select extremely rare compounds with desirable features. With the Extreme Diversity™ platform, Ra is able to incorporate synthetic, non-natural amino acids into peptides by in vitro ribosomal translation and efficiently cyclize the products. Through this approach, Ra can rapidly generate and select high affinity molecules with drug-like properties.
Ra has leveraged the Extreme Diversity™ platform to rapidly develop a portfolio of products with a focus on inhibiting the complement system and other immune targets. Additionally, Ra has an established partnership with Merck focused on interrupting protein-protein interactions to address unmet medical needs across a number of therapeutic areas.
Ra Pharma’s first clinical compound, RA101495, is a peptide inhibitor of complement C5. RA101495 is a potent inhibitor of complement-induced red blood cell lysis and is being developed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).