First-in-Class Small-Molecule Therapeutics
for RNA-Driven Neurodegenerative Diseases
RaritasBio has developed a proprietary small-molecule platform designed to selectively bind and modulate pathogenic RNA repeat structures. Our integrated approach combines unique and novel assay technologies with deep biophysical insights to address unmet medical needs in Huntington's disease and related conditions.
Our technology is engineered to
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Target toxic RNA conformations with high selectivity
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Preserve normal cellular and genetic function
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Enable oral administration and repeat dosing
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Support CNS drug-development requirements
⇒ This platform is designed for translation, not just discovery.
Drug Discovery Platform
Our drug discovery platform integrates medicinal chemistry and biological validation to efficiently advance RNA-targeting small molecules toward clinical readiness.
1. Structure–Activity Relationship (SAR) Optimization
Iterative medicinal chemistry to refine potency, selectivity, and drug-like properties
2. Cellular Validation in Disease-Relevant Models
Lead compounds are evaluated in our disease-relevant cellular models, confirming selectivity
3. Target Engagement & Validation
Confirmation of selective interaction with pathogenic RNA targets
4. Functional Confirmation & Advancement
Consistent activity confirmed to support advancement toward in vivo studies
Together, these steps enable efficient progression from discovery to development-ready therapeutic candidates.
From Huntington’s Disease to a Scalable Platform
Huntington's disease serves as the lead indication for RaritasBio's RNA-targeting platform, which is inherently extensible to other neurodegenerative disorders caused by similar RNA repeat expansions. Success in Huntington's disease enables rapid expansion into related conditions such as myotonic dystrophy and spinocerebellar ataxias, supporting a multi-program pipeline built on a shared technological foundation.