Our innovation:
Development of the new QS, an innovative tool for nucleic acid delivery, allowing the transported nucleic acid to perform its activity inside the cell (cytosol).
The engineered QS are composed by preservatives and sterol derivates, all elements FDA and EMA 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
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)
These QS can be also used as a bioimaging and theranostic tool.
Competitive advantages:
Engineered QS are prepared by a single-step and robust CO2-based methodology that can be scaled up while performed under GMP conditions. CO2-based methodology guarantees superior structural homogeneity, both regarding size and lamellarity, and reproducibility. The nanovesicles are chemically and physically stable over time (several months). QS shown great RNA protection from ribonuclease-mediated degradation. QS are versatile and tuneable drug carrier which can be functionalized either with targeting units or imaging agents obtaining multifunctional nanovesicles.
To be determined during further development.
Patient need addressed: neuroblastoma
Aida Castellanos Paez
