MoleRNA
The platform for designing of small molecules targeting RNA
From RNA Sequencing to Therapeutic Candidate
Our integrated platform combines advanced bioinformatics, biology and chemistry to enable, in three key steps, the validation of mRNA targets and the identification of small molecules binding to selected pockets of RNA. Using state-of-the-art structural and computational approaches, MoleRNA identifies RNA regions with ligand-binding potential and discovers small molecules that modulate biological processes in which the targeted RNA is involved. This approach enables precise regulation of RNA-mediated processes that are relevant to various diseases.
Step 1: Targetable RNA Regions
Identification and characterization of RNA targets
- mRNA isoform analysis in cell lines and patient samples using bioinformatics
- Prediction of stable structures and conserved regions
- Secondary structure validation by SHAPE-MaP in vitro and in cellulo
- Functional annotation of RNA regions using published data, CLIP-seq or RIP method
- Functional validation with ASOs and LNAs
- Biophysical characterization of RNA-protein interactions
- Transcriptome-wide off-target prediction
Step 2: Hit Identification
Screening and validation of active compounds:
- 3D RNA structure determination by modeling, X-ray, or CryoEM
- Hit discovery via virtual screening of RNA-focused libraries or DEL/NMR approaches
- Hit validation through in vitro assays (MST, SPR, AlphaScreen, ITC)
- Selectivity testing against other RNAs
- Protein-level validation in cells (Western Blot, ELISA, reporter assays)
Step 3: Hit Optimization
Development of small-molecule candidates:
- 3D structure resolution of RNA-ligand complexes (X-ray)
- Affinity improvement through structural motif incorporation
- Selectivity optimization toward other RNAs
- IP strategy support and patent portfolio development
- Enhanced cell penetration and bioavailability
- ADME/PK optimization with a focus on oral availability
- Safety profiling in vitro/in silico (hERG, CYPs)
Key Advantages
| Step | Goal | Description |
|---|---|---|
| Step 1 | Accurate target identification | Synergy of bioinformatics, omics and experimental approaches |
| Step 2 | Fast hit discovery | Combination of rapid computational and precise experimental methods |
| Step 3 | Optimized candidates | Comprehensive optimization of affinity, ADME/PK, and safety |
Why MoleRNA?
- Experience: 10+ years in small-molecule development, with two clinical-stage drug candidates
- Technology: Proprietary small-molecule libraries and advanced structural modeling systems
- Infrastructure: Dedicated servers, secure data platforms, full in vitro/in vivo facilities, and equipped chemistry labs
- Partnerships: Collaborations with leading biotech companies and academic institutions worldwide
We provide an innovative platform for discovering small molecules targeting RNA, integrating cutting-edge bio- and chemoinformatics, RNA and cell biology, as well as medicinal chemistry. MoleRNA enables precise discovery of novel RNA ligands, accelerating next-generation therapies. We invite academic groups, biotech, and pharma companies seeking novel drug discovery approaches, as well as RNA researchers looking for project support.
Contact:
Maurycy Zieliński
Bussiness Development Director
m.zielinski@molecure.com
+48 608 500 793
ul. Żwirki i Wigury 101, 02-089 Warszaw, PL