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EXPANSION FOCUS

Expansion focuses on structured RNA targets that are evolutionary conserved or genetically demonstrated to cause disease 

Expanding the Druggable Universe

Expansion’s SMiRNA™ platform identifies druggable RNA structures to deliver oral medicines and transform patients’ lives

1

Utilize patient genetics to identify  disease-driving RNA structure 

3

Utilize structural biology SMiRNA™ platform to identify RNA targeted medicines 

2

Screen proprietary RNA-focused libraries for molecules that modulate RNA targets 

SMiRNA™ Up Close

SMiRNA™ Platform

Utilization of Structural Biology to Advance Pipeline of Oral Small Molecule Drugs

GENERATE

GENERATE pharmacophore model followed by virtual screen and hit expansion

MAP

MAP binding site of SMiRNA™ interaction using high field NMR

UTILIZE

UTILIZE structure based design to inform medicinal chemistry

Star

RNA structures drive disease that can be drugged by orally bioavailable small molecules

  • We define druggable RNA structures by using knowledge of genetics and evolution

  • We utilize proprietary tools and small molecule libraries, Chem-CLIP, NMR /docking /computational tools “RNA specific medicinal chemistry to efficiently identify drug-like small molecules that bind and modulate these structures

  • We solve three-dimensional structures of small molecule-RNA complexes to enable efficient optimization of lead medicines

  • We leverage RNA-specific medicinal chemistry to develop potent and specific compounds

  • We develop target specific biochemical assays and analysis in patient-derived cells

  • Lead compounds are optimized in pre-clinical models to deliver orally bioavailable medicines to treat the whole patient

How We Think about Targeting RNA with Small Molecules.

RNA offers nearly unlimited potential as a target for small molecule chemical probes and lead medicines...

Targeting RNA with Small Molecules to Capture...

The biology of healthy and disease-affected cells is often mediated by RNA structures, desirable...

Precise Small-Molecule Recognition of a Toxic CUG RNA Repeat Expansion.

Excluding the ribosome and riboswitches, developing small molecules that selectively target RNA...

Design of Small Molecules Targeting RNA Structure from Sequence.

The design and discovery of small molecule medicines has largely been focused on a small number of druggable protein...

A curated selection of publications are available here

PUBLICATIONS

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LOCATIONS:

Headquarters

Boston, MA

Research

Jupiter, FL