Expansion Therapeutics
Expansion Therapeutics is a pioneering company focused on developing transformative oral medicines for severe RNA-mediated diseases, including neurological disorders like ALS, Alzheimer's, and Frontotemporal Dementia. Their mission is to address the root causes of these diseases through innovative small molecule drugs that target RNA structures, leveraging advanced scientific research from the Scripps Research Institute and a team of experienced scientists and leadership. The company aims to change patient outcomes by creating effective, orally deliverable therapies that can cross the blood-brain barrier and treat multiple tissues.
Industries
Nr. of Employees
small (1-50)
Expansion Therapeutics
Patents
Products
Preclinical program targeting r(CUG) repeats in DMPK mRNA (Myotonic Dystrophy Type 1)
Small-molecule program designed to bind toxic r(CUG) repeat structures in DMPK pre-mRNA to displace sequestered proteins and restore normal splicing across affected tissues.
Preclinical program targeting G4C2 repeat expansion in C9orf72 (ALS / FTD)
Small-molecule program that binds G4C2 repeat expansion RNA in C9orf72 intron 1 to prevent repeat-associated non-ATG translation and promote correct splicing and decay of toxic RNA.
Preclinical program targeting Tau pre-mRNA splicing (Alzheimer’s disease and FTDP-17)
Small-molecule program aimed at modulating Tau pre-mRNA splicing to address pathogenic splicing patterns linked to Alzheimer’s disease and Frontotemporal Dementia with Parkinsonism.
Preclinical program targeting r(CUG) repeats in DMPK mRNA (Myotonic Dystrophy Type 1)
Small-molecule program designed to bind toxic r(CUG) repeat structures in DMPK pre-mRNA to displace sequestered proteins and restore normal splicing across affected tissues.
Preclinical program targeting G4C2 repeat expansion in C9orf72 (ALS / FTD)
Small-molecule program that binds G4C2 repeat expansion RNA in C9orf72 intron 1 to prevent repeat-associated non-ATG translation and promote correct splicing and decay of toxic RNA.
Preclinical program targeting Tau pre-mRNA splicing (Alzheimer’s disease and FTDP-17)
Small-molecule program aimed at modulating Tau pre-mRNA splicing to address pathogenic splicing patterns linked to Alzheimer’s disease and Frontotemporal Dementia with Parkinsonism.
Services
Acquisition and collaborative development of academic research programs and external assets to expand internal discovery efforts.
Acquisition and collaborative development of academic research programs and external assets to expand internal discovery efforts.
Expertise Areas
- RNA-targeted small-molecule drug discovery
- Computational chemistry and molecular modeling
- Structural biology (NMR) and RNA structural determination
- Biophysical and biochemical assay development
Key Technologies
- High-field NMR spectroscopy
- Molecular dynamics (MD) and QM/MM simulations
- Docking and virtual screening
- Pharmacophore modeling