ARTICLE | Product R&D

Shooting the messenger

Biotechs' reviving interest in mRNA-based protein replacement therapies

January 8, 2015 8:00 AM UTC

Although mRNA-based therapies have caught people's attention in the last few years as vaccines for cancer or infectious diseases, two recent announcements suggest mRNA is gaining traction in protein replacement therapy, too. Silence Therapeutics plc and Arcturus Therapeutics Inc. each have disclosed mRNA therapeutic programs based on delivery systems that produce high yields in animal models and hold promise for low toxicity, but the biggest hurdles will be targeting the relevant tissues and sustaining mRNA levels long enough to create viable therapeutics.The companies join ethris GmbH, Moderna Therapeutics Inc. and BioNTech AG's BioNTech RNA Pharmaceuticals GmbH subsidiary in a race to generate the first mRNA replacement therapy suitable for testing in humans. This week, Moderna announced a $450 million venture financing for its mRNA platform, which includes various forms of mRNA products in addition to protein replacement therapies.

Because mRNA can be used without any modifications to create antigens and activate the immune system, the technology has been used by at least two companies, Argos Therapeutics Inc. and CureVac GmbH,to create clinical stage vaccines for cancer immunotherapy or infectious disease, notably HIV. ...