Our innovation:
Innovative method for the synthesis and encapsulation in an all-in-one process of a functional version of enzymes with high biological (i.e. enzymatic) activity, vehiculized in EVs for protein based therapies.
The nanodelivery system and the therapeutic compound are produced and isolated at the same time in a simpler process than conventional protein purification processes.
The method produces a mixture comprising proteins with a therapeutic interest and EVs or fragments of lipid bilayer of these EVs, both EVs and proteins obtained from the same cell.
In the case of lysosomal enzymes our system increases the catalytic activity of the enzyme with respect to the naked enzyme produced in the same cell, fact that translates into a much higher biological activity in preclinical disease models.
Competitive advantages: Increase of the treatment efficacy while reducing the production cost. Potential transference of this system to other LSD such as Sanfilippo, Gaucher, Pompe, Hurler or Hunter, and other protein based therapies. Application to other diseases currently treated with recombinant proteins.
To be determined during further development.
Patient need addressed: Lysosomal storage diseases (Fabry disease), cancer, infections, immune disorders.
Aida Castellanos Paez
