RNA interference (RNAi) is a mechanism used to defend against external invasion. Oligo- and poly-nucleotide based RNAi therapeutics comprise a high clinical potential for unmet medical diseases and are seen as promising approach to combat a number of different indications including oncology, genetic disorders, auto-immune disorders, orphan diseases and viral infections.
Without a targeting technology, the therapeutics are predominantly metabolized by the liver. To prevent metabolization, the oligo-nucleotides are applied locally, limiting the clinical window for oligonucleotide therapies. Alternatively, targeted delivery to specific cells has to be developed to create novel therapies.
To widen the therapeutic use of oligonucleotides, CIS Pharma is developing a technology for bio-conjugation of RNAi to targeting moieties, similar to the targeting technology developed for cancer therapies. The technology is developed to conjugate both single-stranded antisense oligonucleotides, ASO, as well as double-stranded short interfering RNA, siRNA.
CIS Pharma’s polymer-based delivery system is expected to protect the therapeutic RNA from degradation, to increase circulation time. The carrier is conjugated to targeting moieties.