Expanding small molecule horizons

How 5AM newco Expansion Therapeutics is hitting RNA targets with small molecules

With a platform boasting more than a decade of preclinical rigor and $55.3 million in series A funding, Expansion Therapeutics Inc. is the latest company to step into the RNA-binding small molecule arena. The newco is setting its sights first on pathogenic RNA repeat expansions responsible for a range of rare genetic disorders.

As the most recent graduate of 5AM Ventures’ 4:59 Initiative incubator, Expansion emerged from stealth mode yesterday, after twelve months’ work with scientific founder Matthew Disney, a pioneer in the field who is professor of chemistry at The Scripps Research Institute.

5AM was joined in the series A by Sanofi Ventures, Novartis Venture Fund, Kleiner Perkins, RA Capital Management and Alexandria Venture Investments. Expansion President and CEO Kevin Forrest said that the involvement of corporate VCs reflects emerging interest in the field and that Sanofi Ventures, which joined in the seed round, makes “a great strategic fit” because it is one of the industry leaders in orphan diseases.

The announcement marks the third newco formed in as many years that aims to expand the scope of disease targets druggable by small molecules by developing compounds that bind RNA rather than protein.

Last March, Arrakis Therapeutics Inc. announced a $38 million series A round to

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