Setting Up the Lyophilization of Complex Polyplexes: Study of Sucrose and Trehalose as Lyoprotectants


Serra Vallmitjana, Laia


Lung cancer is one of the deathliest tumors among all neoplasms, accounting for almost 20% of deaths caused by cancer worldwide. Non-small cell lung cancer (NSCLC) is the most common subtype of lung cancer and is usually detected in advanced stages of the disease, which supposes a short life expectancy for the patient. In the last decades, a big part of the scientific research funding has been destined to find a cure for NSCLC. Even though a lot of therapies discovered during these years have cured a lot of people, the disease’s burden and mortality are still substantially high. This situation enlightens the need of more research and new methodologies to find a final solution to this health issue. Gene therapy is a novel technique capable of stopping the growth and replication of cancer cells through the silencing of an oncogene using small interfering RNA (siRNA). This new alternative to traditional cancer therapies has been under deep study in the recent years, mostly because of its ability to personalize the treatment to each patient and the type of cancer diagnosed. However, siRNAs have a very labile nature and cannot be administered alone without suffering degradation. Additionally, a targeting moiety is needed to assure that the treatment acts only on the malignant tissue. Having in mind this context, a group of researchers from IQS Barcelona developed a type of polymeric nanoparticles (NPs) called pBAEs that are able to encapsulate siRNAs, protecting and targeting them in the body. In order to store these NPs, since solid formulations are always more stable than liquid ones, lyophilization was proposed. Nevertheless, the NPs properties should be maintained during this process, and this is one of the most striking points of nanoformulation. In this project, two different polysaccharides (sucrose and trehalose) are studied in order to evaluate their ability to protect the NPs from lyophilization. Trehalose showed more promising results than sucrose, maintaining NP size and polydispersity. However, in terms of cell viability and transfection efficiency, sucrose was more protective. In conclusion, both polysaccharides could partially protect the NPs, but no decisive results were obtained and more studies are needed.



Fornaguera Puigvert, Cristina


IQS SE - Undergraduate Program in Pharmacy