Expansion Therapeutics

Transformative oral medicines for severe RNA-mediated diseases.

About

Founded

2016

Headquarters

San Diego, California

Industry

Healthcare

Company Description

Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases. Expansion has assembled the intellectual property, know-how, and proprietary enabling technologies and tools necessary to facilitate the creation of potent and specific small molecule binders of RNA. Through this unique platform, Expansion is building a portfolio of novel RNA-targeted drug candidates with activity across a broad number of disease indications. The company’s initial development focus is on therapies for patients with expansion repeat diseases who currently have limited and unsatisfactory treatment options.

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Funding

Total Raised

$142.2 million

Valuation

Undisclosed

Significant Investors

Westlake Village BioPartners, Surveyor Capital, Logos Capital, RA Capital Management, 5AM Ventures, Kleiner Perkins, Sanofi Ventures, Novartis Venture Fund

Company Traction

  • As of June 2023, Expansion Therapeutics has completed the discovery phase before pre-clinical trials for Myotonic Dystrophy Type 1, Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD), and Dementias and Movement Disorders.

Leadership Team

  • Renato Skerlj: CEO at Expansion Therapeutics. Founder of X4 Pharmaceuticals.
  • Valerie Cullen: SVP of Research at Expansion Therapeutics. Former VP of Translation and Development at Generian.
  • Mark Ledeboer: SVP of Drug Discovery at Expansion Therapeutics. Former VP of Drug Discovery at Goldfinch Bio.
  • Jennifer Visco: VP of Finance & Business Operations at Expansion Therapeutics. Former Finance Director at Harvard Medical School.
  • Andrew Good: VP of Computational Chemistry at Expansion Therapeutics. Former Senior Director CADD and Cheminformatics at C4 Therapeutics.