Our Pipeline

Pipeline

Disease Areas

Our present areas of focus are halting drug failure caused by resistance mutations in cancer and treating autoimmune and inflammatory diseases.

Cancer

During cancer treatment, mutations that lead to drug resistance and drug failure are common, predictable, and will occur with any kind of targeted therapy. Our synthetic immune lethality approach is a completely original method of using srRNA to prevent known cancer drug resistances from occurring with the goal of significantly extending the time that people with cancer may benefit from a targeted therapy.

Our srRNAs educate the immune system to recognize and remove these mutations as they arise. The hypothesis our trials will test is that administering srRNAs concurrently with targeted therapy will force tumors into a lose-lose situation: As long as the tumor keeps the mutation being targeted by the drug, the drug keeps working to kill the cancer. If the tumor tries to rid itself of the mutation, the immune system springs into action to mount a lethal attack on the cancer. We call this forced choice “synthetic immune lethality.”

Synthetic Immune Lethality

Synthetic Immune Lethality

Immune and Inflammatory Disorders

Inflammatory disease is often driven by molecules expressed systemically in high levels. Current RNA-based approaches are limited by their inability to durably express large quantities of protein modulators that could counteract these inflammatory molecules. Our srRNA vectors are uniquely positioned to be enabling in this space owing to their low doses and durable protein expression. Our current pipeline includes srRNA-encoded molecules that target downstream mediators of the inflammasome and short-circuit the pathways that drive several inflammatory and autoimmune diseases.

Other Conditions

The Replicate srRNA platform has broad potential for applications in other areas such as infectious disease vaccines and gene therapies.