SmartGene.Si Project: Innovative Approaches to Gene Therapy

Gene therapy is a therapeutic strategy in which genes, rather than drugs, are used for treatment. The primary goal is to deliver a functional gene into a target cell, where it acts as a therapeutic agent. Genes are introduced into cells via vectors such as viruses or plasmid DNA. Plasmid DNA is delivered using various techniques, with electroporation being a commonly used method.

Plasmid DNA intended for human gene therapy is produced in Escherichia coli bacteria in compliance with Good Manufacturing Practice (GMP) requirements. Gene therapy begins with the selection of a therapeutic gene, followed by its insertion into a vector, production of the vector, and delivery of the vector to patients. Before human trials, therapeutic genes and vectors must be tested in vitro in cells and in vivo in animal models.

The SmartGene.Si project addresses the challenges of gene therapy – from therapeutic gene selection to production and patient delivery – through innovative, globally applicable solutions. The main goal of the project is the translation of an innovative cancer treatment, developed by the SmartGene.Si consortium, into clinical testing. The therapy will first be evaluated in preclinical studies in small animals and subsequently in a Phase I clinical trial.

A new electroporation device will be developed for the delivery of vectors carrying the therapeutic gene. Additionally, an innovative vector production process will be established and transferred into a new GMP production concept, referred to as “Smart GMP.” All project objectives are based on the know-how and intellectual property of the consortium partners.

The project is partially funded by the European Union, through the European Regional Development Fund (ERDF), and supported by the Slovenian Ministry of Education, Science and Sport. It is carried out under the framework of the Operational Program for the Implementation of the European Cohesion Policy 2014–2020, Priority Axis 1 – International competitiveness of research, innovation, and technological development, with a focus on smart specialization to increase competitiveness and promote a greener economy. Special attention is given to strengthening research infrastructure, developing knowledge and skills, and promoting collaboration between national and European research centers.

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