New EU consortium for new gene therapies EU research project
Gen scissors. Image: Jürgen Fälchle /stock.adobe.com
Scientists at the Ludwig-Maximilians-Universität in Munich are partners in a new research project of the European Commission. Eight international universities will work on nine Working Packages. Over the next five years (until 31 December 2023), the EU provides a total of 15 Mio Euro funding, about 1.1 Mio for the LMU.
Starting date was 1 January 2019 and the kick-off meeting will take place in Milano at 28 February and 1 March 2019, as management and scientific administration are based there.
Besides the Milanese Fondazione Telethon and the LMU, the other partners work at the Universidad Pompeu Fabra, Barcelona (Spain), Universita Degli Studi di Trento (Italy), Technische Universität Dresden (Germany), Vrije Universiteit Brüssel (Belgium), Universite de Nantes (France), and the Massachusetts General Hospital of the Harvard Medical School, Boston (United States).
The working group of Professor Ernst Wagner, Coordinator of the Working Package “Tailor gene-delivery strategies”, and Dr Ulrich Lächelt are investigating the transport of new formulations for gene therapeutic applications, in detail the „viral and non-viral mediated delivery of genes and genome engineering components“. In the focus is the delivery of gene editing tools such as the CRISPR/Cas9 system. “For that purpose, we will apply non-viral vectors, one of our core competences.” Explains Wagner.
The other Working Packages also benefit from the expertise of renowned scientists. For instance, Luigi Naldini, General Coordinator of the Consortium and working at the Telethon in Mailand, is a gene therapy specialist, and Keith Joung, Massachusetts General Hospital in Boston, is an expert in the field of gene editing.
Gene therapy has recently shown remarkable potential to offer definitive treatments for otherwise incurable diseases. Currently, seven gene therapy products have reached the market and many more are entering clinical testing for selected indications.
Moreover, emerging technologies for targeted gene editing are complementing the scope of conventional gene transfer, opening the way to precise gene correction and making possible to silence, activate or recode any sequence of interest in the genome. However, in order to realize the full potential of these strategies and broaden application of gene therapy, the field has to solve several major hurdles.
UPGRADE will exploit and further develop disruptive new technologies for precision gene and epigenome editing and for site-specific transgene insertion. The safety and efficacy profile of each advanced medicinal product (AMP) will be stringently validated in tissues (hematopoiesis, heart and skeletal muscle, liver, retina) and disease models (muscle wasting, storage and blood disorders, hypercholesterolemia).
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