Technology

We have developed a unique and patented RNA Therapeutic platform that leverages novel peptide technology that is able to systemically target and deliver the RNA therapeutic into the pathologic cell to fight or prevent diseases.

 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 (RRRTM) 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., KRASG12C), 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.