The unique features of InteRNA’s differentiating R&D approach:
- miRNA Library: Use of next generation sequencing and bioinformatics to create a broader microRNA library
- Screening: Non-biased (agnostic) phenotypic functional screening in cell-based assays (instead of descriptive expression profiling)
- Chemical Modifications: Application of chemical modification to enrich uptake of active strand in the RNA-Induced Silencing Complex (RISC)
- Delivery: Use of next-generation lipid nanoparticle (LNP) formulation selected on localized cellular delivery/delivery beyond the liver, endosomal escape, and pharmacodynamic effects
- MoA: Use of RNAseq, Ingenuity Pathway Analysis software, and bioinformatics to decipher molecular/biological Mode of Action (MoA)
- IP: Unique IP portfolio around drug candidates and formulation
Beyond oncology, we have shown this R&D approach qualifies also for the identification of miRNA drug candidates for treatment of Central Nervous System (CNS) indications, e.g., mesial Temporal Lobe Epilepsy (mTLE) and neurodegenerative disease (see Scientific Publications).