BY OUR PLATFORMS
BY THERAPEUTIC AREA
01 October 2022 - 30 September 2027
Start – End dates: 01/10/2022 – 30/09/2027
Project reference: 101070903
Total budget: 3,912,835.00€
Financed by: European Union (Horizon EIC Grants, Pathfinder Challenges, Emerging Technologies in Cell and Gene therapy)
“This project has received funding from the European Union’s Horizon Europe research and innovation programme under grant agreement No 101070903”
Partners: Institut de la Vision/Sorbonne University (France); Museum National d’Histoire Naturelle/Institut National de la Sante et de la Recherche Medicaleinserm (France); Imagine Institut des Maladies Genetiques Necker Enfants Malades Fondation (France); Erasmus Universitair Medisch Centrum Rotterdam (Netherlands).
Genome editing technologies based on CRISPR/Cas systems allow targeted genomic modification with unprecedented precision and have emerged as powerful alternatives to the conventional gene therapy approaches for various human diseases, with a series of clinical trials in progress. However, some crucial challenges remain to be addressed to enhance efficiency and safety and decrease the costs of treatments.
Current viral-based delivery systems are associated with a high risk of toxicity and immunogenicity and remain highly expensive. We will develop a new generation of non-viral delivery systems for gene editing tools based on the use of modified nanoparticles with human-derived protein moieties that will allow targeting the tissue and cells of interest in vivo with minimal adverse effects.
Prime editors have raised exciting possibilities for double-strand break-free genome editing. However, a major limitation of current prime editors is highly variable efficiency both from one target to another and between cell types. We will design and evaluate novel prime editor tools in order to both increase activity per se and overcome cell-specific limitations.
We will test our approach on the hematopoietic system to treat Sickle Cell Disease, avoiding the challenges and risks of hematopoietic stem cell manipulation associated with current gene therapy approaches, and thus providing a treatment much simpler, safer, and cost-effective to implement.
Our technological breakthroughs address two key obstacles in cell and gene therapy: gene editing efficiency and systemic delivery. The novel prime editors and targeted nanoparticles that we will engineer will be combined to make unprecedented off-the-shelf, recombinant biologics for gene therapy. The versatility of the design of these novel recombinant biologics makes them suitable for the treatment of a vast majority of genetic diseases.
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