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A Spanish biomedical research center has developed an innovative method for the synthesis of a functional version of enzymes with high biological activity (i.e. enzymatic). They have reached to this method by encapsulation in an all-in-one process, vehiculating these enzymes in extracellular vesicles (EVs). This method can be applied for protein-based therapies, and the research center is looking to establish license, research cooperation or technical cooperation agreements for this technology.
This Spanish research networking center gathers some of the most important Spanish research groups in biomedicine, located in more than 100 institutions like universities, hospitals, and technological centers distributed around the country.
The biomedical use of proteins (i.e., enzymes, growth factors, hormones) has been widely explored by the biotechnological industry including enzyme replacement therapy (ERT).
One of the major bottlenecks regarding therapeutic protein treatments, such as those used in ERT to treat lysosomal storage diseases (LSD), it is to achieve a proper biodistribution and biological availability of the protein at the right tissues and organs. Conventional approaches tend to administer high doses of the protein product to obtain the desired therapeutic activity, which often leads to deleterious side effects.
The use of nanotechnology to deliver active proteins might overcome this particular concern by increasing protein stability, reducing clearance and directing the protein to specific cells. Particularly, the use of extracellular vesicles (EVs), as vehicles for biologically active proteins. EVs have been shown to be biocompatible and fully tunable, allowing the production and encapsulation of recombinant proteins inside in a single step.
The research center has generated specific IPR on the use of EV as protein delivery vehicles and tested several examples such as EVs loaded with the lysosomal enzyme alpha-Galactosidase (GLA). EVs-GLA exhibited significantly higher enzymatic activity than the reference enzymatic treatment in vitro and in vivo and proved to be an ideal system for ERT in lysosomal storage diseases.
The research center is looking to establish license, research cooperation or technical cooperation agreements with industrial partners, mainly from the Biotech and the Pharma sectors, for entering into preclinical R&D collaboration or proof of concept development.
These new enzymes with high biological activity can be used in the treatment of lysosomal storage diseases (LSD) as fabry disease, or in cancer, infections, or immune disorders.
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 the 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, the fact that translates into a much higher biological activity in preclinical disease models.
The system increases 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.
In vitro studies, ready for in vivo studies
Priority European patent application filed (January 24, 2019)
Suitable for international extension (PCT application)
Patent ready for licensing-out.
The research center is seeking industrial partners, either Biotech or Pharma, to:
- enter into preclinical R&D collaboration (in vitro and/or in vivo drug testing).
- Further development until clinical proof-of-concept