nir-vana innovation network

The delivery of exogenous nucleic acids into eukaryotic cells is called transfection and is essential for cell and molecular biology research and biotechnology. It is also widely regarded as a promising approach for the treatment of genetic diseases and cancer.

Chemical vectors are an improved alternative to physical and viral methods of transfection, mainly because they are safer, cheaper, more reproducible, and do not present significant DNA size limits. To date however transfection efficiency has been their main limitation.

The newly developed reagent consists of a polymeric material based on repeating squaramide compounds. These polymers strongly bind and fold polyanionic compounds such as nucleic acids (DNA and RNA) in a wide range of sizes. This packaging protects nucleic acids from degradation and facilitates delivery across biological membranes. This, combined with the effective release of nucleic acids within cells, greatly increases the efficiency of transfection in a wide variety of organisms.

The developed polymer outperforms commercially available chemical vectors, and has a wider field of use. It is effective in organisms where existing reagents or methodologies perform poorly or simply do not work. Moreover, the results obtained in gene editing, virus reconstitution and in vivo transfection assays highlight its potential for developing future biomedical applications.

This reagent has been developed as a multi-centre project (Spain and UK), involving researchers with expertise in the areas of chemical synthesis, biochemistry, molecular biology and parasitology. The polymer, as a product for transfecting cells, is in the pre-commercialisation phase. The team is currently collaborating with different companies and academic collaborators to validate the product in a range of specific biomedical applications.

The team aims to expand collaborations with other companies in the healthcare sector, where transfection efficiency currently limits biomedical research and medical applications. Companies interested in testing the product will be offered a material transfer and confidentiality agreement, with subsequent negotiation of a licensing agreement if the compound proves to be superior to their current methodology.

Advantages & innovations

The main advantage of this transfection reagent is that it can be applied to a broad spectrum of cell cultures of all types, including those classified as difficult to transfect. It therefore opens the door to transfection of organisms for which no alternative transfection methods currently exist. The transfection vector – aka pASQUA, is obtained by polycondensation in a single synthetic step from commercial products and is water soluble, chemically stable at room temperature, resistant to hydrolysis, easy to handle and store. Its production is easily scalable. It packages macromolecules (DNA, RNA and peptides) regardless of their size and structure (including circular and linear nucleic acids). Cell transfection is carried out following a simple two-step protocol and it has been determined that entry of the packaged material into the cell interior occurs very quickly: 30 minutes is sufficient to obtain excellent transfection efficiency. The transfection reagent is less cytotoxic than the standard methods used to transfect a wide range of cell types.

Stage of development

Prototype available for demonstration

Partner sought

The main goal is to explore and characterize the reagent as a chemical vector with biomedical applications. To this end, the research team are looking for companies interested in evaluating the potential of new transfection agents to improve their experimental outcomes. A two-phase collaboration is offered: a first phase of testing and validation of the product, non-commercial, under a material transfer and data confidentiality agreement; and a second phase of commercial use of the product. On the assumption that the results of the first phase are satisfactory, this would lead to a royalty-bearing licensing agreement.