RNA Therapies Start-Up Raises $55.3M in Early Funds

RNA molecule illustration (Nicolle Rager Fuller, National Science Foundation)

3 January 2018. A start-up company creating treatments for inherited diseases that target faulty RNA transcribed from genetic codes is raising $55.3 million in its first venture funding round. Expansion Therapeutics Inc. in San Diego is a spin-off enterprise from the lab of Scripps Research Institute chemistry professor Matthew Disney, scientific founder of the company.

Expansion Therapeutics discovers and develops small molecule, or low molecular weight, drugs to treat diseases caused by ribonucleic acid, or RNA molecules that either result from mutations, or incorrectly transcribe DNA genetic codes into protein instructions for cells. Recent research on RNA, says the company, shows the molecules form structures and patterns that provide targets for therapies, which the company plans to exploit. The first of these disease targets are expansion repeat disorders, where repeated short sequences known as microsatellites expand beyond a safe length, stopping genes from functioning or causing aberrant proteins to be produced.

The company says at least 30 disease conditions can be traced to  expansion repeat disorders. The first of these disorders addressed by Expansion Therapeutics is type 1 mytotonic dystrophy, caused by defects in the DMPK gene. Another, less severe form of the disease, known as type 2, results from defects in a different (CNBP) gene. Symptoms of type 1 mytotonic dystrophy include myotonia — inability of contracting muscles to relax — progressive muscle weakness, and malfunctioning central nervous, cardiac, and endocrine systems. Myotonic Dystrophy Foundation says the condition affects about 1 in 8,000 people worldwide, including some 40,000 individuals in the U.S.

Disney’s lab at the Scripps Research Institute facility in Jupiter, Florida studies RNA molecular structures and behavior, discovering targets and potential therapies. Expansion Therapeutics licenses technologies from Scripps for identifying and developing small molecule drugs that bind to these expansion repeat disorder targets, beginning with type 1 mytotonic dystrophy. The company says it is also identifying treatments for type 2 mytotonic dystrophy and two other undisclosed diseases.

“It is clear,” says Disney in a company statement, “that disease-related RNA is now an addressable target with small molecule medicines and we are now on the verge of developing treatments for patients with the most urgent medical needs. We will work tirelessly to fulfill this promise.”

In its first round of venture finance, Expansion Therapeutics is raising $55.3 million led by investment companies 5AM Ventures and Kleiner Perkins, as well as the venture capital arms of pharmaceutical companies Novartis and Sanofi.  RA Capital Management and Alexandria Venture Investments are also taking part.

5AM Ventures, an investor in early-stage life science enterprises in Boston and San Francisco, is a founder of Expansion Therapeutics, which also incubated the company at its 4:59 Initiative. Both 5AM Ventures and Sanofi Ventures provided seed funding for the company.

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