Our Approach & Strategy
RNA has recently ascended as an exciting therapeutic class. Current mRNA technologies, however, face numerous and ongoing challenges to production, delivery, and performance that have prevented the field from reaching its full potential. Lacking caps or tails and having greater cellular persistence, circular RNA had the potential to address these challenges. However, the technology to create and engineer them at will in a manner that was also scalable was absent – until now.
Inspired by RNA enzymes and RNA viruses, Orna now possesses this technology – harnessing sophisticated molecular innovations to advance a system for production, delivery, and performance that is superior to conventional long RNA approaches. oRNAs are easy to make, are always full length, and are well-tolerated inside cells creating a simple, powerful platform: the next breakthrough in RNA therapeutics. From the design of oRNA to delivery, Orna carefully optimizes each step for superior product performance in the body.
Orna has built a complete platform to create and industrialize oRNA technology, capturing attractive features that are not possible with traditional RNA therapeutics.
PRODUCTION – oRNAs are engineered as linear RNAs that undergo highly-efficient autocatalytic circularization. Proprietary purification methods complete the production loop.
EXPRESSION – Orna utilizes proprietary IRES technology to maximize protein output.
DELIVERY – Orna uses its well-rounded capabilities in RNA formulation to adapt lipid nanoparticle (LNP) technologies to create uniquely-formulated, optimally delivered oRNA that safely reaches the site where it is needed in the body.
A NEW WAY TO ADDRESS DISEASE
The potential applications of Orna’s technology are far reaching, including cancer, autoimmune diseases, regenerative medicine, and genetic disorders. Orna is currently evaluating where our medicines can make the biggest difference, with an initial focus on treating challenging diseases where other modalities have fallen short. As an initial focus, Orna believes it can apply its oRNA technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (in situ CAR therapy).