The current landscape of gene therapy, while holding immense promise for curing severe genetic diseases, is heavily constrained by the reliance on traditional viral vectors. These vectors pose significant challenges, including safety risks such as immunogenicity and the potential for insertional mutagenesis, alongside difficulties in achieving cost-effective, large-scale manufacturing. These limitations ultimately restrict the broad and safe application of life-changing curative treatments for patients.
The FICAT (Find-Cut And-Transfer) project is driven by the ambition to establish a new paradigm and radically change this landscape. The overall objective is to develop and validate a novel, highly efficient, and non-viral Gene Editing System that completely bypasses the safety issues and manufacturing constraints of viral vectors. FICAT utilizes a cutting-edge non-viral delivery system based on lipid nanoparticles (LNPs) combined with a proprietary, highly efficient integrase enzyme. This unique combination is engineered to achieve stable, long-lasting gene expression in target cells, overcoming the major drawback of transient gene expression seen in many other non-viral methods.
FICAT's pathway to impact is designed to be transformative. By offering a safer, non-immunogenic and scalable platform, the project aims to significantly lower the production costs of gene therapies, thereby enhancing global accessibility for patients and sustainability for healthcare systems. Our initial focus is a first-in-human clinical demonstration for a liver disorder, which will serve to validate the platform's universal potential for treating a wide array of monogenic diseases. This work directly addresses a strategic European Union priority: the urgent need for faster, safer and more affordable advanced therapeutic medicinal products (ATMPs).
