This technology describes compositions which enhance the potency and stability of nucleic acid therapeutics for the treatment of diseases or conditions.
In recent years, numerous nucleic acid therapeutics have been described for the treatment of a myriad of diseases and conditions. Several of these drugs have been approved by the FDA, including the first small interfering RNA (siRNA) and micro RNA (miRNA)-based drugs. However, the success of these nucleic acid therapeutics remains limited by factors including off target effects, the limited half-life of RNA molecules, and unstable payload expression and release.
This technology describes several compositions which improve the performance of miRNA-based therapeutics using a co-delivery platform in which the therapeutic is paired with a stabilizing or enhancing element. This element may be an RNA induced silencing complex (RISC) protein, to stabilize payloads within target cells, thereby maximizing therapeutic effectiveness. The technology uses exosomes or other co-delivery vehicles which are engineered to minimize endogenous nucleic acid loading, to reduce off-target effects and enable targeted therapeutic delivery.
This technology has been validated with human cancer cell lines and in mouse models.
Fatemeh Momen-Heravi, DDS, MPH, PhD, MS
IR CU21087, CU20006
Licensing Contact: Joan Martinez