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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

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Utilize structural biology SMiRNA™ platform to identify RNA targeted medicines 

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

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Utilize patient genetics to identify  disease-driving RNA structure 

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

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

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  • 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

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

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

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

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  • 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...

Full Paper

A curated selection of publications are available here

PUBLICATIONS

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