RNA replaces enzymes
How Moderna replaced ERT with RNA for a rare metabolic disease
Moderna Therapeutics Inc. is continuing to build its pipeline of RNA alternatives to enzyme replacement therapies with a compound to treat the rare metabolic disease MMA. The goal is to provide more predictable kinetics and side-step the liver toxicity of DNA gene therapies.
Last month, Moderna published preclinical data in Cell Reports on MRNA-3704, an mRNA encoding the metabolic enzyme methylmalonyl CoA mutase (MUT) absent in