The company is seeking funding to initiate company operations for execution of a gene silencing protocol in the Huntington’s disease model. Outcomes of this study will validate commercial viability of the technology and open additional targets in trinucleotide repeat disorders.
OBJECTIVE FOR FUNDING
Demonstration of gene silencing and cell uptake of CNA oligomers in an initial treatment model for a trinucleotide repeat disorder. Objectives: (i) Demonstrate synthesis of a repeating compound using a single step click reaction as proposed in company intellectual property, (ii) demonstrate biologic function of the compound by inhibition of the reporter associated with the Huntington’s gene.