Upstart Expansion bags a $55M launch round to join the hunt for RNA-targeting small molecules

Arrakis isn’t the only biotech startup looking to develop RNA-targeted small molecule drugs. Now they have a rival in Expansion Therapeutics, a San Diego-based biotech with R&D roots in the lab of Scripps’ Matthew Disney, who’s had a longtime interest exploring the field.

Expansion is having a coming out party this morning, celebrating a substantial $55.3 million launch round from some A-list investors: 5AM Ventures and Kleiner Perkins led the round, with help from Novartis Venture Fund, and Sanofi Ventures as well as participation from RA Capital Management and Alexandria Venture Investments.

Kevin Forrest

The company was seeded by 5AM and Sanofi Ventures and incubated in 5AM’s 4:59 Initiative.

Kevin Forrest, a former 5AM principal who went on to help launch San Diego-based Cidara with Jeff Stein, has the CEO job at the new venture. It’s a platform tech with a variety of applications, but Forrest says the initial focus is on expansion repeat disorders, with a set of 30 genetic diseases they could go after.

That includes myotonic dystrophy type 1, or DM1, one of the most common types of adult onset muscular dystrophies, says Forrest, who studied RNA for his PhD.

Arrakis’ team under Michael Gilman and Russ Petter — which lined up new IP from Penn’s David Chenoweth just weeks ago — may have been the first to emerge from stealth mode looking to do this, with an underground operation dating back to 2015, but Forrest argues that Disney (no relation to the famous Disneys) and his colleagues in their Jupiter, FL lab were out there first, with the largest body of successful preclinical work to boast about.

Matthew Disney

“I would argue that Matt’s the clear scientific leader,” says Forrest, citing the scientist’s published work in peer reviewed journals.

That may be somewhat academic at this point, though, as both companies have a ways to go before they can begin to start testing their drugs in humans. But Arrakis hasn’t overlooked the same field of rare genetic conditions that attracted Expansion, listing them as one of several top R&D focuses.

Forrest steadfastly declines to even suggest how long it could be before their first treatment will start a human study, or how long the round will last.

Right now the staff is about 12 people, after 5AM and Sanofi seeded it for the first year of its existence. You can double that number with senior advisers, says the CEO, and you can look for the staff to just about double over the next year.

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