Small RNA
Self Delivery
RNA editing cargo is small enough to get into cells without the need of viral or non-viral approaches. It can be formulated in solution and chemically modified to protect it from degradation. Further chemical modifications enhance its stability and extend the half-life of the cargo. This approach is not a one and done and we expect dosing would need to happen several times a year. Alternatively, small RNAs can also be delivered with a medical device for continuous delivery. Micro pumps exist for intracranial or intrathecal delivery which can manage a sustained concentration in spinal fluid.
Importantly, small RNAs have been used clinically for many years with much success. Because of their chemical synthesis and small size, small RNAs can advance rapidly.
