This technology identifies steric-blocking antisense oligonucleotides (ASOs) that target an indispensable noncoding region of MYC mRNA for the treatment of MYC-dependent cancers.
MYC is dysregulated in approximately 70% of human cancers. Although MYC is a promising target for cancer therapy because of its central role in tumor initiation, progression, and maintenance across a wide range of cancer types, there are currently no FDA-approved direct MYC inhibitors on the market. Therapeutic targeting of MYC protein has remained challenging due to its intrinsically disordered structure that lacks well-defined druggable domains, and nuclear localization which creates a barrier for effective drug delivery.
This technology is a set of steric-blocking antisense oligonucleotides (ASOs) that bind to a critical region within the 3’ untranslated region (3’UTR) of MYC mRNA. These ASOs bind and block cytoplasmic MYC mRNA function without inducing its degradation. Targeting this regulatory element disrupts MYC mRNA localization and impairs nuclear import of MYC protein, thereby suppressing MYC-dependent transcriptional activity. As a result, these ASOs can inhibit cancer cell growth without detectable toxicity.
This technology has been validated in human cancer cell lines, mouse models, and patient-derived tumor organoids.
Imetelstat, an FDA-approved oligonucleotide telomerase inhibitor for lower-risk myelodysplastic syndromes, provides clinical precedent for the feasibility of oligonucleotide-based therapeutics in cancer.
Patent Pending
IR CU25384
Licensing Contact: Joan Martinez
