OUR PROPRIETARY CONDITIONALLY ACTIVATED siRNA (CASi) PLATFORM COMBINES ADVANTAGEOUS PROPERTIES OF SINGLE AND DOUBLE-STRANDED RNA IN A SINGLE MOLECULE, ALLOWING FOR EFFICIENT SELF-DELIVERY AND UPTAKE, POTENT AND DURABLE KNOCKDOWN. THE PLATFORM ALSO OFFERS THE ABILITY TO TARGET CELL TYPES AND CELL STATES, AN UNPRECEDENTED APPROACH THAT CAN REVOLUTIONIZE THE WAY WE THINK OF TACKLING THESE DEVASTATING DISEASES.
THE CASi CONSTRUCT
EACH CASi MOLECULE CONSISTS OF A MODULAR SENSOR DOMAIN AND A PAYLOAD DOMAIN, SUCH AS A SMALL INTERFERING RNA (siRNA).
The addition of the sensor strand gives the CASi expanded chemical and structural space, allowing for:
The combination of a unique mechanism for effective self-delivery as well as cell selectivity, enables new avenues for next generation RNAi therapy.
The unique ability to target RNAi activity to specific cell populations with unique expression of sensor genes. By enabling cell selective knockdown of targets by RNAi, CASi has the ability to improve the therapeutic window for existing targets and open the door for numerous, therapeutic applications.
We are an emerging precision neuroscience biotechnology company pioneering a new type of RNAi medicine, with the goal of treating a range of CNS diseases.