Building next generation RNA therapeutics for genetic medicine and beyond

circVec is Circio´s differentiated, proprietary technology for in situ circRNA production in cells. circVec enables enhanced and more durable vector protein expression than conventional mRNA-based systems, which can enable more potent gene therapies.

The circVec platform is a modular genetic cassette designed for efficient biogenesis of multifunctional circRNAs in human cells. circVec can be tailored for any DNA or viral vector system, enabling protein expression via circRNA. The modular design allows for the incorporation of a variety of functionalities into the circRNA, opening the opportunity to create highly targeted medicines with potential applications in a variety of therapeutic settings ranging from genetic disease to vaccines, oncology and cell therapy.

 

Primary development area:

Gene therapy – AAV-circVec

Rare diseases caused by specific genetic defects are a major unmet medical need of increasing focus in drug development. Current gold-standard gene therapy is based on Adeno-Associated Virus – AAV – vectors. Although AAV vectors have been the most successful gene therapy approach to date with six products approved, AAVs suffer from relatively low expression level, toxicity and high cost. Circio is deploying its circVec system in AAVs to enhance the protein expression level and imporve durability. Current data suggests that circVec has the potential to improve the potency of AAV gene therapy by 10-100x, which could enable reduced dosing, lower cost and a better safety profile, as well as opening up new disease areas to AAV gene therapy. In parallel, Circio is exploring a variety of other DNA and viral vectors to broaden the platform and its potential targets.

 

Additional opportunities:

Oncology – circAde

Circio is the first company to make significant advances in the delivery of circRNA to solid tumors. The circAde approach is based on Circio´s clinically validated adenoviral vector system, with first generation mRNA vector ONCOS-102 having demonstrated strong immune responses and clinical efficacy in several solid tumor types. Learning from the broad clinical and translational experience with ONCOS-102, the circAde vector system has been engineered for robust in situ expression of circRNA directly in cancer cells with multiple parallel functionalities for optimal anti-cancer and immune-stimulatory activity.

Vaccines – circVac

circRNA allows for prolonged antigen expression, making them highly attractive for development of vaccines against infectious disease and cancer. circVac is our proprietary vector-based platform for antigen expression and robust immune stimulation.  circVac vaccines are expected to stimulate more durable immune responses than current mRNA-based approaches, and offer important advantages for vaccine handling and dosing.