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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
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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
A curated selection of publications are available here
PUBLICATIONS