Delivery of RNAi: A novel approach for delivery of Nucleic Acids to Cells
Provides a solution to the rate-limiting step in the delivery of RNA-based drugs (RNAi, miRNA, mRNA).
Although, RNAi and antisense technologies have the potential to be developed as targeted therapeutics, delivery of these nucleic acid molecules has been a challenge.
Problem to be solved:
Nearly 3 decades of research and pharmaceutical/drug development attempts with these types of molecules have failed due to this singular challenge. Extensive chemical modifications to enhance cellular delivery of these drugs as well as the development of nanoparticle delivery systems have met with limited success have involved extensive chemical modifications that have resulted in several undesirable outcomes such as; loss of biological activity, increased toxicity, rapid clearance, unwanted pharmacodynamics etc.
Several hundreds of million dollars have been expended in trying to realize the promise of nucleic acid drugs. The ability to deliver nucleic acids to cells will enable development of therapies for many unmet clinical needs, with an estimated market in the billions. Ability to target these drugs to specific cell types further enhances its potential opportunities far beyond what is currently possible.
Veritas Bio Solution:
Veritas Bio’s technology utilizes a delivery/communication system that already exists in humans and animals. On a regular basis, our cells extrude vesicles that are composed of cellular membranes and some cellular components derived from the mother cell. The components include nucleic acids and proteins. The extruded vesicles travel via blood or lymph to distal sites, into organs, tissues and cells, where their cargo is delivered, released and taken up in functional form by other cells. The technology utilizes these carrier vesicles to carry small and large exogenous nucleic acids (antisense, RNAi, miRNA, mRNA) into cells. Furthermore, these carrier vesicles can be modified to include molecules on its surface to enable cell specific targeting.
Since a natural process is being harnessed, the need for costly and toxic delivery systems is abolished.