The Potential of RNAi

RNA Interference (RNAi) is a recently discovered natural biological process. The Central Dogma of biology is that DNA makes RNA, and RNA subsequently makes protein. Because undesired proteins are the cause of most human disease, pharmaceutical drugs typically target select proteins and block their function. RNAi works upstream from the manufacture of protein in cells, silencing genes and thereby blocking the creation of these disease-causing proteins before they are made.

This breakthrough discovery is being harnessed by RNAi researchers to develop an entirely new class of human therapeutic that could potentially treat sixty percent of all human disease – the Interfering RNA. This new class of drugs brings with it enormous potential:

• Significantly improved specificity of target molecules
• Greater efficacy with fewer side effects
• New drugs for rare or difficult to treat diseases
• Reduced drug discovery timelines
• Faster response to pandemic infection

Learn about Traversa's RNAi Delivery Technology: PTD-DRBD 

RNAi Delivery Problem

Interfering RNAs have tremendous selectivity, degrade only target RNAs, and yield specific gene silencing. However, due to their relatively large size (~14,000-18,000 Daltons), they require an additional delivery technology in order to enter cells and produce their intended effect. This ability to deliver interfering RNA into the cell is critical to the success of the technology. RNAi delivery technologies are, perhaps, as important to the safety and efficacy of the commercialized RNAi therapeutic as the siRNA warhead itself.

Solving the RNAi Delivery Problem

Delivery is the one significant remaining problem to be solved before RNAi can achieve success as a drug class, and Traversa's technology solves this problem.

Traversa's siRNA delivery technology targets 100% of all cells assayed in a non-cytotoxic fashion, including: primary cells, human stem cells, hematopoietic lineages (T cells, B cells, Macrophage) and tumor cells, where other approaches have failed. Traversa is also focusing efforts on incorporation of cell specific targeting domains and enhanced Protein Transduction delivery based on the mechanism of action.