RNA can instruct your body to make customized, highly efficacious medicines, with few side effects and at low cost.
Synthetic Genomics has engineered a proprietary RNA replicon platform to selectively stimulate the immune system and deliver targeted therapies produced within a person’s own body. Our RNA platform overcomes common shortcomings of traditional replicon and messenger RNA (mRNA) technologies, enabling a new class of vaccines and combination therapies to combat cancer and infectious diseases.
RNA replicons use the body’s cellular machinery to produce ample amounts of proteins that can trigger a protective immune response (to prevent the flu, for example) or produce a therapeutic response to treat diseases like cancer. Our RNA replicons are powered by an “engine” that carries specific instructions to cells to replicate RNA. The body’s natural machinery takes over from there, quickly converting the genetic instructions into thousands of copies of RNA. The RNA replicon is translated to produce the desired antigens or other proteins that trigger the body’s immune response or deliver a direct therapeutic effect.
Conventional development and manufacturing of protein-based medicines requires costly and complex manufacturing processes. Our RNA replicon is a synthetic platform that has the potential to undercut expensive vaccine and therapeutic production costs — eliminating the need for large manufacturing facilities. In addition, the technology is tunable, or adjustable, so it can be quickly adapted to respond to mutations in disease-causing pathogens or to incorporate new therapeutic molecules.
How Our RNA Replicon Is Different
Bypass inflammation-mediated shutdown
Inflammation-mediated shutdown suppresses vaccine or therapeutic efficacy and can be observed with traditional mRNA-based approaches. Cells stop producing proteins in response to either sterile inflammation, which can be triggered by chemotherapy, radiation, immune-therapies, high-fat diets, obesity or imbalanced microbiota, or infectious inflammation, which is often seen in people with viral, bacterial or parasitic co-infections. Thus, inflammation is an underlying condition in many patient populations, which can curtail the utility of other RNA-based therapeutic platforms. Our proprietary RNA replicon is designed to specifically bypass this mechanism — but without preventing cells from promoting inflammation, which is important for driving immune response.
Superior in vivo protein expression
mRNA has a rapid peak (roughly 24 hours) and decay (signal lost in 5-7 days). By contrast, our RNA replicon platform is capable of durable expression beyond 28 days for therapeutic proteins. Once administered into a cell host, our engineered RNA replicon generates tens of thousands of copies that direct the production of protein within hours.
Multigenic and tunable
Traditional vaccines are limited to expressing a single antigen to trigger immune response. When multiple proteins are included, this has traditionally required separate manufacturing processes for each protein. Our RNA replicons can express multiple antigens simultaneously, with the potential to trigger a broader, more effective immune response. Furthermore, each gene is individually controllable, allowing customization for individual pathogens, diseases, or therapeutic protein production. This is important for balancing ratios of heavy and light chains for antibody expression, or for avoiding toxicity when co-encoding immunostimulatory molecules in vaccines.
Superior immune responses
Conventional or modified mRNAs are poor inducers of T cell responses. Our engineered RNA replicon platform, however, increases the magnitude and quality of acute and memory T cell responses. It produces strong, protective antibody responses and holistically engages the immune system, which is critical in complex infectious disease and cancers.
Cost-effective, scalable, and rapid manufacturing
Affordable and rapid manufacturing enables a fast response to emerging disease threats. With RNA replicons, a single-liter bioreactor can produce more than five million doses, allowing for a tiny GMP manufacturing footprint when compared with traditional technologies. The self-amplifying nature of our RNA replicon platform results in a high-potency product (projected human vaccine doses of one microgram or less) that can be produced at pennies per dose and has an increased safety profile. Importantly, the fully synthetic manufacturing allows for vaccine production within weeks instead of months. Our approach is highly scalable, allowing production, purification, and formulation on a personalized scale (milligram quantities) or at scales compatible with global distribution (gram quantities).