Protease Assisted Native-Protein Delivery Approach (PANDA)

Researchers at UCLA have developed a novel technology to safely and efficiently deliver native proteins to target cells for therapeutic, imaging, tissue engineering and regenerative medicine purposes.

Recombinant protein based drugs represent a very promising avenue of therapy for a number of medical applications and the market for protein therapeutics is currently projected to reach $141.5 billion by 2017. Despite their great commercial success, many of these drugs suffer from significant obstacles in the areas of delivery. To date, a number of protein delivery approaches have been pursued including electroporations, microinjections, protein transduction domain (PTD)-mediated platforms, and noncovalent methods. Though promising, these methods suffer from various limitations that make them clinically unfeasible. The ability to deliver protein products in an efficient and safe manner would be a significant achievement that could potentially open up an entirely new avenue of medical technologies for clinical use.

Researchers at UCLA have developed a novel protein delivery approach termed the Protease-Assisted Native-protein Delivery Approach (PANDA). This technology is centered around the use of protease-degradable nanocapsules to coat native proteins for delivery into cells. Upon uptake of these complexes into target cells, the nanocapsules are degraded by cellular proteases, allowing the protein cargo to be released. The ease of preparation, high cell penetration capability, and long-term stability associated with this system, coupled with its low toxicity and protease-modulated specific degradability, make it a versatile and very promising candidate for use in the delivery of therapeutic agents.

Extensive in vitro experiments validating the technology have been performed. Studies further characterizing application of this technology to other proteins and in vivo proof-of-concept experiments are underway.