Remix Therapeutics debuts with $81M to 'reprogram' disease-causing RNA

Drugs normally target problematic proteins, but Peter Smith, Ph.D., believes his team at Remix Therapeutics can disrupt the process of a protein’s creation before it starts causing disease. Investors believe in him, too—the company launches with $81 million in funding to develop small molecules that reprogram RNA processing.

It’s possible to interrupt the process where harmful genes are expressed, said Smith, co-founder, president and chief scientific officer of Remix Therapeutics and an entrepreneur in residence at Atlas Venture.

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Rather than fix a mutation that codes for a disease-causing protein, Remix is taking aim at cell components that process RNA, a molecule that copies instructions in DNA and acts as the blueprint for protein synthesis. With its oral small molecules, it hopes to fine-tune how new RNA sequences are produced—intervening before they're translated into proteins—and treat a variety of diseases, such as cancers, central nervous system disorders and other rare diseases. 

Remix Therapeutics spent the past year developing its REMaster technology, which provides a foundation for developing drug candidates. The platform, according to Smith, includes three main components: a database of messenger RNA molecules called transcriptomes as potential drug targets, screening technologies to either validate genetic targets or discover and optimize a chemical map, and a proprietary chemical library to serve as a starting point for drug discovery.

Targeting RNA may serve as the future of medicine, but its complexities have made it difficult to drug in the past.

“I think what we're really doing is leveraging our insights into the cellular complexes that control RNA processing on the outside systems that we've built,” Smith said. “By using those complexes in the physiologically relevant settings, I think it really gives us a great head start on the kind of targets that we know are important.” 

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The launch provides the foundation for building the company’s platform and eventually getting therapies into clinical trials, said Remix's chief business and chief operating officer, Heather Wasserman.

It’s also planning to add to its 18-strong team.

“The bulk of our hiring has been scientists and we will continue to build out in chemistry, data science and then in RNA biology,” Wasserman said. 

Atlas Venture and The Column Group provided $16 million in seed financing, joining a series A round of $65 million led by Foresite Capital. 

“I think what's been really exciting as we’ve built this platform is the breadth of targets and disease areas that we can see insights towards,” Smith said. “We've narrowed that focus over the last 12 months we've got a collection of targets and projects that we're really excited about.”