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

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