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Huntington’s Diseases Therapy
Competitive advantages:
GENE THERAPIES for HDCAG sh-miRNATechnologyRNAiActing on the mutant gene✓Targets 100% HD patients✓Long-term silencing✓Universal therapy for other diseases with CAG expansion✓
GENE THERAPIES for HDExon-targetingTechnologyRNAiActing on the mutant gene✗Targets 100% HD patients✓Long-term silencing✓Universal therapy for other diseases with CAG expansion✓
GENE THERAPIES for HDSNP- targetingTechnologyAntisenseActing on the mutant gene✓Targets 100% HD patients✗Long-term silencing✗Universal therapy for other diseases with CAG expansion✗
About Huntington’s Disease Therapy
Huntington’s disease therapy – interfering RNAs are designed to target directly the CAG repeat tract. Specific modifications of their sequence result in formation of mismatches between interfering RNA and a target transcript. Typical normal huntingtin transcript binds a single RISC, whereas the mutant variant binds more silencing complexes. During protein synthesis, the mismatched RISC from normal transcripts is easily depleted in contrast to the multiple RISCs on the mutant transcripts, which are stable and inhibit translation.
This leads to allele-selective inhibition of mutant huntingtin synthesis. The degree of selectivity depends on the difference in length between the repeat tracts in the normal and mutant alleles.
Our novel RNAi therapy will bring a long-term silencing effect of the mutated gene and therefore will inhibit the progression of neurodegenerative diseases caused by a toxic protein produced by CAG repeats.