nir-vana innovation network

This research networking center gathers some of the main Spanish research groups in biomedicine, located in more than 100 institutions as universities, hospitals and technological centers distributed around the country.

RNA therapeutics is an emerging field with a promising number of targets around all the transcriptome, which includes small RNAs like small interfering RNA (siRNA), microRNA (miRNA), among others. Although, the RNA based-therapies may be an alternative to chemoresistant tumors, the in vivo administration is still a challenge in the field, due to the rapid clearance and degradation of small RNAs in the bloodstream.

As of today, nanomedicine promises the precise delivery of drugs to disease sites (i.e. tumours) with reduced off-target toxicities. Liposomal nanovesicles are one of the most promising candidates for drug delivery in nanomedicine. However, these liposomal-based medicinal products still present unsolved drawbacks such as poor colloidal stability, short half-life when administered in the blood circulation, nonspecific bio-distribution and modification of endogenous RNA machinery.

Novel engineered non-liposomal nanovesicles (QS) have been developed to overcome some of the previously stated problems related to application of RNA based therapeutics in the treatment of human diseases, such as cancer.

Advantages & innovations

The new QS is an innovative tool for nucleic acid delivery, allowing the transported nucleic acid to perform its activity inside the cell (cytosol). QS may be used in the development of RNA (Ribonucleic acid) based therapeutics for the treatment of human diseases, such as cancer. The engineered QS are composed by preservatives and sterol derivates, all elements FDA (U.S. Food and Drug Administration) and EMA (European Medicines Agency) approved. These QS have shown great in vitro transfection efficiencies and good biocompatibility, comparable to other commercial transfection reagents (e.g. Lipofectamine® 2000) and also potent anti-tumoural activity in neuroblastoma cell lines. Development of a pharmaceutical composition comprising a therapeutically effective amount of the nanovesicle and a pharmaceutically acceptable excipient or vehicle can be carried out. The nanovesicle or the pharmaceutical composition can be used as a medicament for the treatment of diseases that use nucleic acids as therapeutic agent (e.g. cancer, in neuroblastoma) These QS can be also used as a bioimaging and theranostic tool.

Stage of development

Under development/lab tested

Partner sought

Patent ready for licensing-out. The research center is seeking industrial partners, either from the Biotech or Pharma sectors, to: - enter into preclinical R&D collaboration (in vitro and/or in vivo drug testing) through research cooperation agreements. - Further development until clinical proof-of-concept via license or technical cooperation agreements.