Ctgct En V2

CTGCT, a center to bridge the gap between research and therapeutic solutions

The Center for the Technologies of Gene and Cell Therapies was funded in September 2024, after the NIC received a grant and the mission to start a project that would focus on creating a pipeline of biomedical tools focused on the development of therapeutic solutions for rare genetic diseases and difficult-to-treat cancers. The CTGCT is an infrastructure center within the NIC and a European Center of Excellence (CoE). The project is funded by the European Union, UK Research and Innovation and the Ministry of Higher Education, Science and Innovation of Slovenia. Project: 101059842 – CTGCT – HORIZON-WIDERA-2022

 

mission & vision

Mission: The CTGCT aims to be a pioneering research institution in Slovenia, addressing critical healthcare challenges through cutting-edge research in cancer and rare genetic diseases. We co-create medical breakthroughs by bridging innovative research with clinical practice, working alongside renowned institutions. By transferring knowledge through international collaborations and striving for scientific excellence, we enable the advancement of gene and cell therapies globally, with a special focus on the Western Balkans and Eastern Europe.

Committed to excellence, inclusivity, and collaboration, we want to attract top talent and Slovenian scientists working abroad while fostering a stimulating environment for young researchers. The mission of CTGCT is based on translating scientific achievements into tangible therapeutic applications that serve society and stand as a beacon of innovation in the world. While providing ground-breaking research, the center will continue to work intensively on building public trust in science, demonstrating how vision, dedication, and high-quality work lead to meaningful healthcare outcomes that transform patients’ lives.

 

 

Vision: We push the boundaries of gene and cell therapy science with cutting-edge research in cancer and rare genetic diseases with a focus on neurodevelopmental diseases in compliance with Good Manufacturing Practices (GMP) standards. We co-create innovative therapeutic solutions for the healthcare challenges of the future. We enable breakthrough discoveries and career development by transferring knowledge to young scientists and striving for good mutual relations with clinicians, regulatory authorities, and industry partners. Committed to scientific excellence and ethical research practices, we want to transform global healthcare and stand as a leading force in gene and cell therapy.

Laboratories of

the CTGCT

The center will bring together important pieces of research and analytical equipment that researchers and clinicians will have access to. For an infrastructure center, it is important that the equipment is top-notch and regularly maintained.

Premises for the production of reagents for clinical testing according to GMP guidelines

The cGMP facility will produce viruses, nucleic acids, and non-viral delivery reagents for clinical therapy, supported by storage and waste management areas, with key equipment including single-use bioreactors, downstream processing, and purification systems

Laboratory for performing quality analysis of GMP products

The quality control unit will be adjacent to GMP rooms with basic and advanced analytical equipment including a flow cytometer, dd/qPCR, and sterility tester for required product quality analyses.

Laboratory for preclinical research

Testing on experimental mice will follow GLP guidelines using breeding, surgery, and analysis equipment plus advanced devices like MRI/CT imaging, and microscopy systems.

Laboratory for the development of new technologies

 A GLP-compliant support laboratory will handle preclinical R&D with reduced cleanliness requirements but comprehensive equipment for cell, virus, and nucleic acid work including bioreactors, cell sorters, and analytical systems.

With excellent people we will create excellent results

Prof Dr Roman Jerala

Prof Dr Roman Jerala

Head of the Department of Synthetic Biology and Immunology, National Institute of Chemistry

Innovative use of synthetic biology for the programming of molecules and cells for improved efficacy and safety of advanced treatments

Roman is the Head of the Department of Synthetic Biology and Immunology, where, with a large group of motivated collaborators, covers the range from atoms to organisms. The focus of research is on the development of new biomedical technologies and the translation of biomedical discoveries for advanced therapies. They entered the field of synthetic biology through extremely successful student research projects at the iGEM competition, which began at MIT with meetings of teams from all over the world.

Mojcabenčina Original

Prof Dr MOJCA BENČINA

Head of the Infrastructure Center, National Institute of Chemistry

Knowledge and innovation hand in hand for advances in medicine

Long-time collaborator at the Department of Synthetic Biology and Immunology, where she leads a team of research colleagues in the field of ultrasound applications in conjunction with synthetic biology and immunology to regulate molecular biological cell processes, and an infrastructure group for the rendering of cell processes by fluorescent techniques. She teaches intellectual property protection at the Biotechnical Faculty and Microbiology at the higher education Institute Physiotherapy. She is an official representative for the protection of industrial property rights at the European and the Slovenian patent office. She is the recipient of several prestigious awards, such as: Zois Award for Outstanding Achievements in Immunology and Synthetic Biology, Pregl’s Award for Exceptional Scientific Achievements in Chemistry and Related Disciplines, “Socrates Award for High School Didactics” (SOVA) and the award Prometheus Science for Excellence in Communication.

Dlainšček

DR Duško lainšček

National Institute of Chemistry

Expert in the field of genome modification

Duško is a leading Slovenian expert in genome modification using the CRISPR/Cas system also with the aim of creating immunotherapy cell products. He has extensive experience in the study of therapeutic applications in vivo.

DR MIHA MODIC

National Institute of Chemistry and UCL/KCL

Development of synthetic RNA biology tools for advanced gene therapy

Miha is Sir Henry Wellcome Senior Fellow (UCL/KCL London), working between the Francis Crick Institute, EMBL, and The National Institute of Chemistry. He is developing novel systems biology approaches and functional RNA biology tools for improving the specificity of gene and cell therapies.

IN COLLABORATION WITH OUR PARTNERS OF EXCELLENCE

UNIVERSITY COLLEGE LONDON (UCL)

Dr JANE KINGHORN

University College London

Director, Translational Research Office

She has extensive experience in developing therapies (gene & cell, small molecules, antibodies, oligos) to clinical benefit gained from over 25 years working in large pharma and academia. Her experience has spanned the drug discovery & biomarker pathway, leading projects through target validation, lead optimisation, candidate selection through to the clinic. Jane as Director of the Translational Research Office (TRO) at UCL works at the interface between academia, NHS and Industry. She has extensive experience of differing organisational cultures and a deep understanding of the life sciences research & innovation ecosystem in the UK/EU. The TRO comprises a team of 22 highly experienced applied scientists supporting academics deliver novel therapeutics, including advanced therapies, diagnostics and devices. The TRO are instrumental in developing the organisational translational culture, capability and processes necessary to deliver the UCL and the NIHR funded Biomedical Research Centre’s mission of “Accelerating translation for health and wealth”. Embracing diversity and working in partnership is key to this vision.

Dr PAMELA TRANTER

University College London

Head, Translational Research Group

She joined UCL Translational Research Office (TRO) in 2014 and has been leading the Translational Research group since 2016. The TRO collaborates with academics, industry partners and external funding bodies, to facilitate the translation of UCL’s emerging research into therapies and medical products. Pamela is the project manager for substantial late pre-clinical/early clinical advanced therapy projects, with a particular emphasis on CAR T cells and Rare diseases. The TRO has supported the translation of more than 100 active projects securing public funding to the value of greater than £150 Million. Projects are nurtured from preclinical activity into the clinic through to securing VC investment with 5 advanced therapy spin-outs having IPO’d on the Nasdaq (securing >$770M investment). Prior to joining UCL, Pamela gained extensive experience of drug discovery in the pharmaceutical industry at Novartis/Ciba-Geigy where she led small molecule and biologics projects for respiratory and thrombosis indications.

C0125996 Giampietro Schiavo Researcher Meeting N

Prof Dr Giampietro Schiavo FMedSci FRSB

University College London

Deputy Director of the Queen Square Institute of Neurology

Gipi, Professor of Cellular Neuroscience, is Deputy Director of the Queen Square Institute of Neurology, UK Dementia Research Institute Investigator and academic lead of the ARUK Drug Discovery Institute at UCL. He has a long-term interest in the mechanism of entry of neurotrophic factors and their receptors at synapses and their recruitment to signalling endosomes moving along the axonal retrograde transport pathway. This essential trafficking route, which delivers many organelles and molecular complexes to the neuronal soma, is impaired in several human pathologies, such as motor neuron and Alzheimer’s disease. The current focus of Gipi’s team is the identification of novel pharmacological nodes to restore axonal transport in human and rodent disease models.

Schorge Stephanie

Prof Dr Stephanie Schorge

University College London

Head of the Department of Neuroscience, Physiology and Pharmacology at UCL

Stephanie Schorge is the Sophia Jex-Blake Professor of Physiology and head of the Department of Neuroscience, Physiology and Pharmacology at UCL. Her research focuses on how manipulating neuronal excitability with gene therapy can be used to relieve neurological disorders, including non-genetic epilepsies. She is currently leading an MRC-DPFS funded programme to bring gene therapy to a first-in-human clinical trial for refractory epilepsy. Professor Schorge and collaborators at UCL have developed a portfolio of gene therapy approaches and are co-founders of a new UCL Spinout company to bring these to clinical trials.

The UNIVERSITY MEDICAL CENTRE UTRECHT (UMC Utrecht)

JurgenkuballProf Dr Jürgen Kuball

The University Medical Centre Utrecht

He chairs the Department of Hematology at the UMC Utrecht Cancer Centre. Clinical activities focus around acute leukemia and allogeneic stem cell transplantation and he is medical director of the stem cell transplantation program. His research activities focus around tumor immunology and the ATMP development of a next generation of engineered immune cells. Within this context he is scientific co-founder of the UMC Utrecht spin-off company GADETA and holder of multiple patents dealing with engineered immune cells. Kuball is also active member of EBMT and chairs since 2019 the Legal Regulatory Affairs Committee (LRAC) of EBMT.

Technische Universität Dresden (TU DRESDEN)

Ezio

Prof Dr Ezio Bonifacio

Technische Universität Dresden

He is Professor at the Technische Universität Dresden and has been actively working in the area of immune therapy for type 1 diabetes for over 30 years. He has been instrumental in developing, harmonizing and applying measurements of autoimmunity to understand the pathogenesis of type 1 diabetes and to aid in the disease prediction. In 1999, he became Director of the Beta cell replacement programme in Milan with a strong emphasis on cell therapy. He moved to Dresden in 2007 to help develop the diabetes cell therapy programme. He has experience in clinical translation of cell therapy, has established large scale European screening and prevention trials through the Global Platform for the Prevention of Diabetes (GPPAD), is the Speaker for one of the four German DFG Next Generation Sequencing Competence and most relevant to the role in this application he is the Speaker of the German Cluster4Future on cell and gene therapy SaxoCell, which aims to develop this are as an experimental, clinical and industry strength in Saxony.

CHARITÉ Universitätsmedizin Berlin (CHARITÉ)

Prof. Petra Reinke Bih Charité

PROF DR Petra Reinke

Charité Universitätsmedizin Berlin

She is Professor of Internal Medicine, Nephrology and Transplantation at Charité – Universitätsmedizin Berlin and was Medical Director of Nephrology and Transplantation and Head of the Kidney Transplant Outpatient Clinic. She is the founding director of the Berlin Center for Advanced Therapies (BeCAT) and head of the GMP Facility for developing new T-cell-based therapeutic strategies. She is one of the founding members of the (BIH)-Berlin-(Brandenburg) Centre for Regenerative Therapies (BCRT), was a member of the steering committee 2006 – 2021 and vice speaker in 2021. From 2019 – 2021 she was CART Coordinator (Charité) of the European University Hospital Alliance (EUHA). Prof. Reinke has broad experience in leading and coordinating international consortia. She is currently coordinator of the EU HORIZON 2020 “ReSHAPE” consortium, the EU HORIZON-HLTH-2021 (HORIZON-RIA) “geneTIGA” consortium and was in the past coordinator of the EU FP7 consortium “BIO-DrIM” (2012-2018). She was also steering committee member and work package leader in the EU FP7 consortium “ONE- Study” (2010-2015), in the EU FP6 consortium “RISET” (2005-2010), co-PI of an Immune Tolerance Network project (ITN) (2000-2003) and of a ROTF grant (1997-2000). She is a programme board member of the UK Regenerative Medicine Platform of the Medical Research Council (MRC, UKRMP) and a member of several editorial boards of various scientific journals and has published more than 300 peer reviewed articles.

Where to find us

We are part of the National Institute of Chemistry

Hajdrihova ulica 19, 1000 Ljubljana, Slovenia