Gene therapy is a therapeutic strategy using genes as therapeutic agents/drugs. The main goal of gene therapy is to insert a functional gene that plays the role of drug into the targeted cell to cure a disease or to repair a dysfunction caused by a genetic defect. Gene is delivered to the cells in form of vectors such as viruses or plasmid DNA. Plasmid DNA is delivered to the cells by different techniques and electroporation is commonly used to achieve this goal. Plasmid DNA intended for gene therapy applications in humans is produced in bacteria Escherichia coli under Good Manufacturing Practice (GMP) regulations. Gene therapy therefore starts with selection of therapeutic gene, continues with insertion of this gene into the vector, which is followed by production of vector containing therapeutic gene, and delivering vector to patients. Project SmartGene.Si will address gene therapy challenges from therapeutic gene, to production and delivery to patients with innovative globally applicable solutions.
The main objective of the project is translation of innovative cancer treatment, already developed by SmartGene.Si consortium partner from research into clinical trial. Gene therapy treatment evaluated in vivo in preclinical phase will be further tested on pet animals and taken to Phase I of clinical trial. Novel electroporation device to deliver the vector with therapeutic gene will be developed. To facilitate and enable gene therapy trials innovative integrated manufacturing process of gene therapy vector will be developed and transferred to novel “Smart” GMP manufacturing facility, based on new concept. All objectives are based on SmartGene.Si consortium partners know-how and proprietary IP.