The Approaches

Tumor Suppressor Rescue:  Tumor suppressor mutations and deletions resulting in loss of function or inactivation can be drivers of a large percent of human cancers. To date there are very limited treatments to replace, restore and rescue (RRR^TM) tumor suppressor function as a means to reverse the disease.  We have developed a new potent strategy combining proprietary mRNA with a tumor selective peptide-based nanocarrier based on proprietary amphipathic peptides to rescue tumor suppressor function as a potential therapeutic approach in cancers. This approach has resulted in strong inhibition of tumor growth.

Oncogene Editing/Deletion: Specific mutations in oncogenes are often key drivers in numerous cancers. While small molecule approaches have been developed and targeted to specific mutations (e.g., KRAS^G12C), they are often hampered by development of adaptive resistance, mainly associated with the emergence of other mutations.  We have developed a new potent strategy combining gene editing machinery with a tumor selective peptide-based nanocarrier, to impair cancer cell proliferation by directly targeting specific mutations resulting deletion/editing of the mutant oncogene. This approach has resulted in strong inhibition of tumor growth with very few treatments.

Other genetic diseases:  Enzyme and protein deficiency diseases are a major cause of morbidity and mortality. Targeting these diseases with the appropriate mRNA and a peptide based nanocarrier allows re-expression of the delinquent protein and restores normal function. In other situations where overexpression of certain proteins may drive disease, a gene editing/deletion approach using the peptide based nanocarrier can be an efficient approach to normalization.