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Project Factsheet ClickGene Click Chemistry for Future Gene Therapies to Benefit Citizens, Researchers and Industry Background Gene therapy is expected to play a key role in next-generation medicine by correcting the underlying genetic causes of disease, thereby facilitating personalised medicine. As this technology can address a wide range of medical conditions, along with finding substantive application within plant genetics, extensive growth in this sector of the biopharmaceutical and agricultural industry are widely predicted. Thus, to enhance the human understanding and application of gene therapy, highly qualified experts of this field are urgently required. Furthermore, current gene therapy methods possess undesirable side effects, including insertional mutagenesis, toxicity, low efficiency and off-target cutting. Questions also remain regarding the optimal methods for delivering nucleases into cells and tissues. These limitations will be addressed through the original and innovative approach of the ClickGene network, which will provide training to 14 PhD students in an exciting academia-industry collaboration. Objectives ClickGene’s main research objective is to develop new materials and methodologies for site-selective genetic engineering by training researchers within an innovative, multi-dis- Funding Programme: ciplinary and entrepreneurial environment so that they can meet the challenges facing future scientific leaders in Europe’s industry. More specifically, the research of this project will: •• Provide world-class training to 14 PhD students in the areas of synthetic click chemistry, nanotechnology, liposomal drug delivery and nucleic acid (DNA and RNA) chemistry •• Generate scientific excellence and technological innovation in the area of gene Horizon 2020 Framework Programme of the European Union silencing and epigenetic base detection Project Duration: In particular, the European expertise presently available in DNA-based chemical and 01/01/2015-31/12/2018 biotechnology fields needs to be combined to provide young researchers with the appropriate knowledge and know-how to address the gap associated with rapid scientific Project Budget: developments in epigenetics. ClickGene drives efforts to establish multidisciplinary and 3.7 million euro methodologically strong environments in order to foster scientific leaders of our future. Project Website: www.clickgene.eu 2015 © accelopment AG 1 Project Factsheet Activities ClickGene’s research is based on four work packages and three main scientific objectives: Gene therapeutics, diagnostic genome sequencing, and liposomal drug delivery. Gene Therapeutics Peakdale Molecular Metabion DCU Ireland LMU Germany LipiNutraMed LipiNutragen Oxford UK INN Greece Materials for Genome Editing Diagnostic Genome Sequencing us Ind try ademic Ac ISOF Italy WP1: BaseClick ATDBio Link Technologies IOCB Czech Republic WP2: Customised Liposomal Transfection Overview: > More than 10 industry-academia partnerships > World leading scientists > Pioneering industrial collaborators > Radical new therapeutic development > Applicaitons within personalised medicine > State-of-the-art training and research facilites Liposomal Drug Delivery WP3: Gene Knockout: Mechanisms, Activation and Control Innovation: > Future of modern medicine > Targeted therapy for cancer > Targeted therapy for genetic disease > Nano-technology and materials chemistry > Unique training evironments > Critical thinking / problem-based learning WP4: Applications: Biotechnological and Medicinal Evaluation Leadership: > Training future EU scientific leaders > ESRs develop transferable skills > Two secondment periods within industry > Unique dissemination methods > Strong overlap with COST Action Networks > Entrepreneurial development skills Impact Project Coordinator: Dr Andrew Kellett, Dublin City University, IE ClickGene will educate the next generation of biomaterials chemists with specific ex- [email protected] pertise in click chemistry and cutting edge application areas of gene therapy, nanotechnology and biological diagnostics. 14 researchers will emerge with unique skill sets and multi-disciplinary training from leading academic and industrial environments. This will be achieved through individualised career guidance, applied learning, industry and academic networking, close collaboration with COST, a framework to implement new research with interdisciplinary vision, as well as through the integration of non-academic Project participants: •• Ludwig Maximilian University Munich, DE •• Oxford University, UK •• National Center for Scientific beneficiaries including leading SMEs that are linked to ClickGene's partner universities. Research “Demokritos”, GR The results of ClickGene will have an impact on the general public, on industry and on •• National Research Council, IT •• Institute of Organic Chemistry future research: •• The general public will benefit of new gene therapies, drug delivery and biodiagnostics for personalised medicine. •• A new generation of scientists with new sets of techniques to combine will be cru- and Biochemistry ASCR, CZ •• ATDbio Ltd, UK •• baseclick GmbH, DE •• LipiNutraGen srl, IT cial for future research in click chemistry, gene therapy and biological diagnostics. •• A new generation of chemists will boost the European chemical and biotechnology and agricultural industries. 2015 © accelopment AG Subcontractor: •• accelopment AG, CH 2