To include your compound in the COVID-19 Resource Center, submit it here.

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

Read the full 654 word article

Trial Subscription

Get a two-week free trial subscription to BioCentury

SIGN UP

Article Purchase

This article may not be distributed to non-subscribers
More Info >PURCHASE