Expansion Therapeutics develops oral small-molecule medicines that target structured, disease-driving RNA using its proprietary SMiRNA™ platform. The company combines patient genetics and deep informatic analysis to identify RNA structures amenable to ligand binding, screens against RNA-focused libraries, and applies structural biology to design lead-like chemical matter. Expansion is advancing programs toward clinical candidates in myotonic dystrophy type 1 (DM1) and multiple neurodegenerative indications including ALS, FTD and tauopathies. Proceeds from the recent financing will be used to accelerate the SMiRNA platform and identify clinical candidates across these indications. Expansion recently in-licensed two research programs from Scripps Research, including a tau-targeting program tied to dementia disorders. The company is headquartered in Boston with a research facility in Jupiter, Florida, and its scientific founder and colleagues received an American Chemical Society award for RNA-targeting work. Expansion Therapeutics focuses on the discovery and development of RNA-targeted small molecule medicines based on foundational research from Matthew D. Disney’s laboratory at The Scripps Research Institute. The company is building a portfolio of novel small-molecule RNA binders with an initial clinical focus on expansion repeat disorders, a group of roughly 30 genetic diseases including myotonic dystrophy type I. Proceeds from the recent financing will advance Expansion’s pipeline toward treatments intended to address both peripheral and central symptoms of these RNA-driven diseases. Expansion was incubated within 5AM Ventures’ 4:59 Initiative and was seed funded by 5AM and Sanofi Ventures, and it holds exclusive worldwide rights to Disney’s platform technologies and enabling tools. The company has assembled a board and a scientific advisory board of leaders in RNA chemistry, folding, and structural biology and is led by newly appointed president and CEO Kevin M. Forrest. Expansion is based in San Diego, California and Jupiter, Florida.

Total raised

$135M

Funding rounds

2

Latest round

Series B

Latest activity

Sep 2021