This Challenge aims to develop a human relevant risk assessment system that will improve the safety of novel gene therapy products and reduce the reliance on animal models. The winning team is led by GeneWerk and includes Brunel University London, University College London, King’s College London and The Natural and Medical Sciences Institute, at the University of Tübingen, with GlaxoSmithKline and Novartis acting as Sponsors.
They have already set up the basis of a standardised assay for IndividualisedGenotoxicity testing (InGeTox) to assess vector safety and efficacy using human induced pluripotent stem cells. Vector-related factors that cause genotoxicity will be measured, including effects on cancer gene expression by vector promoters and enhancers, aberrant transcript forms or gene splicing, host epigenetic responses to gene transfer and therapeutic gene expression.Find out more about CRACK IT
SCIDNET is a European Commission Horizon 2020 -funded project which started in January 2016. During 4 years the project aims to develop gene therapy for Severe Combined Immuno Deficiencies (SCID) to a medicinal product that can cure affected children. SCID result in severe abnormalities of the immune system, and prevent children to fight against infections. This leads to death in the first year of life if no effective treatment is given.
Together with in total 12 partners across Europe GeneWerk is part of this challenging project. As workpackage leader of WP6 “Genomic integration analysis for SCID gene therapy” our aim is to develop new strategies for demonstrating and monitoring the safety and efficiency of the gene therapy regimens. The initiative lead by Professor Bobby Gaspar (UCL ICH/GOSH) benefits from the excellent expertise of all partners and the funding from the European Commission and we all hope that it will result in a safe treatment for all affected patients.
GeneWerk is a partner of RECOMB – an EU-funded research consortium that aims to develop new treatment options for SCID (see above). The researches’ focus is on developing gene therapy approaches for the treatment of RAG-SCID in clinical trials.
Furthermore, the gained expertise through the cooperation between the RECOMB partners will also be of direct use for the treatment of other diseases with autologous HSC gene therapy. The program will make gene therapy a realistic option for about 70% of all SCID patients. For further information please have a look at the following leaflet: http://www.recomb.eu/resources/leaflet/.
Recomb, now in video – Gene therapies for SCID
Recomb is excited to announce the release of a 2-minute animation and interview video that explains what Severe Combined Immunodeficiency (SCID) is, what the Recomb project is about and its medical and societal impact.
We have built a German AAV partnership called “ABC of AAV”, including Plasmidfactory, Progen, Sirion and GeneWerk. The aim of this consortium is to provide combined capabilities in AAV development, engineering, production and determination of all 4 companies, to provide a one-stop-shop for customers.
The ABC of AAV service offering includes safety evaluation, impurity detection, quantification, plasmid manufacture, ITR integrity, particle engineering and quantification, pre-clinical AAV production and GMP manufacturing services.
GeneWerK’s journey began long before the company was officially founded in 2014 by Prof. Dr. Christof von Kalle, Dr. Manfred Schmidt and Dr. Annette Deichmann, together with German Cancer Research Center (DKFZ).
Today, we are a team of >30 employees providing expertise and services to clients around the world from Heidelberg, Germany.View our history