REMODEL- pREcise gene delivery for advanced disease MODELling

A cutting-edge gene-editing platform to generate complex disease models to test innovative therapeutic approaches.

Start – End dates: 01/08/2021 – 01/08/2023

Project reference: INNOTEC, ACE034/21/000094

Total budget: 199,899.89€

Co-financed by: ACCIÓ, Spain

Partners: SYNBIO-UPF, Barcelona

Summary:

The increasing understanding of the importance of animal welfare in biomedical research has uncovered the need to develop alternative models fulfilling the 3R principles (Replacement, Reduction, Refinement). In this context, ZeClinics has recently developed a leading platform (ZeGenesis) to generate zebrafish disease models to support the pharmaceutical industry in its drug-discovery pipelines. By employing cutting-edge technologies (CRISPR/Cas9 or transposons), the company can modify the zebrafish genome to establish models to test novel drug candidates or validate gene-therapy approaches. However, it is highly challenging to achieve, at the same time, highly efficient and precise targeted gene delivery for germline engineering. In collaboration with Translational Synthetic Biology group of Pompeu Fabra University and their new technology FiCAT (licensed to Integra Therapeutics), we will leverage the precision of CRISPR, the efficiency of transposases and the versatility of the zebrafish model to develop a cutting-edge gene-editing platform to generate complex disease models to test innovative therapeutic approaches.