The company develops RNA‑targeting small‑molecule medicines for diseases that lack druggable protein targets, such as certain cancers, lung cancer driven by SMARCA2/4, and neurodegenerative disorders with repeat expansions.
Funding
Funding not disclosed
Founders
Product
Problem
Many diseases lack druggable protein targets because the underlying pathology is driven by RNA, such as cancers with undruggable transcription factors, lung cancers dependent on SMARCA2/4, and neurodegenerative disorders caused by repeat expansions. Traditional small‑molecule approaches focus on protein pockets and cannot directly modulate these RNA-driven mechanisms.
Solution
Endura Therapeutics uses its TRANSCEND platform to discover RNA‑targeting small‑molecule medicines. The platform combines covalent chemistry with next‑generation sequencing to assess each compound’s binding across the entire cellular RNA pool, generating single‑nucleotide‑resolution interaction maps. These maps guide iterative medicinal chemistry, enabling the design of molecules that bind specific RNA sequences or structures implicated in disease. By quantifying RNA binding events in cells, the approach transforms RNA from an “undruggable” target into a tractable therapeutic space, aiming to treat cancers and neurodegenerative disorders that are inaccessible to protein‑centric drugs.
Target Audience
Primary customers are pharmaceutical and biotech companies seeking to develop therapies for diseases driven by undruggable RNA targets, including oncology and neurodegeneration programs.
Features
- Covalent chemistry library designed to form irreversible bonds with RNA nucleotides, increasing binding specificity and potency
- Chemo‑genomics workflow that simultaneously tests each compound against all cellular RNAs using high‑throughput next‑generation sequencing
- Single‑nucleotide resolution interaction maps that reveal precise binding sites and off‑target profiles
- Data‑driven hit optimization powered by AI algorithms that prioritize compounds with favorable RNA selectivity and drug‑like properties
- Scalable platform capable of generating massive interaction datasets without the need for individual target assays