Immunostimulatory RNA Therapeutic for Treatment of Infectious Disease and Cancer

The Problem

The COVID-19 pandemic has made it painfully clear that the world lacks effective drugs that can prevent or treat respiratory infections quickly. Rather than developing specific drugs to treat individual pathogens, it would be much more efficient to create a therapy that could activate the body’s innate immune system – our first line of defense against both pathogens and cancers – to treat a broad range of health threats. However, the innate system is largely driven by the action of interferons (IFNs), which can cause deadly cytokine storms and other risks. Therapeutics that can safely target the innate immune system would offer an entirely new “disease-agnostic” approach to prevent and treat a broad range of diseases.

Our Solution

Wyss researchers have discovered a new class of immunostimulatory double-stranded RNAs (dsRNAs) that produce pan-antiviral and anti-cancer activity by inducing the expression of multiple types of IFNs. dsRNAs can effectively inhibit infection of human lung cells by a wide range of viruses including SARS-CoV-2, influenza A, and even common cold viruses, and reduced SARS-CoV-2 infection in a mouse COVID model by up to five orders of magnitude. They also have potent anti-cancer effects, as daily treatment resulted in significant inhibition of tumor growth in a mouse melanoma model. Importantly, dsRNAs do not cause an increase in pro-inflammatory cytokines, making them safer for humans.

These novel dsRNAs could be used both to prevent infection as well to treat patients after they have been infected, and may be particularly useful when administered via inhalation or intranasally. They can also be used to treat bacterial, fungal, and parasitic infections. In addition, they could serve as an adjuvant to enhance the efficacy of existing vaccines. For cancer indications, these novel dsRNAs would simply be administered to patients intravenously.

Project Journey

First discovered during efforts to find an effective treatment for COVID-19 during the pandemic, dsRNAs are being advanced at the Wyss Institute for both infectious disease and cancer indications. From a commercial perspective, these molecules are short (20-27 base pairs), fully chemically defined, and easily synthesized, making them easy to manufacture commercially. By acting to enhance generic host immune responses independent of disease-specific mechanisms, these drugs could represent an entirely new class of innate immuno­therapeutics for a broad range of diseases. This is a 2023-2024 Validation Project.

Several patents covering this technology are currently pending.