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Address: Otfried-Müller-Straße 10
72076 Tübingen


Founding Director

frontend.sr-only_#{element.icon}: +49 7071 29-82168
Prof. Dr. Julia Skokowa


frontend.sr-only_#{element.icon}: julia.skokowa@med.uni-tuebingen.de


Scientific coordinator

frontend.sr-only_#{element.icon}: +49 7071 29-86013
Dr. Olga Klimenkova


frontend.sr-only_#{element.icon}: Olga.Klimenkova@med.uni-tuebingen.de


Gene and RNA Therapy Center (GRTC)

About us

The mission of the GRTC is to develop innovative, personalized, and broadly applicable Gene and RNA therapy approaches that are safe and affordable. By initiating and executing clinical gene and RNA therapy studies, we aspire to offer new perspectives to patients with rare diseases and target common diseases.

Aim of GRTC and collaborating partners

The unique combination of excellent basic, clinical and translational expertise at the Gene and RNA Therapy Center in Tübingen, along with the close collaborations with the MPI of Developmental Biology in Tübingen on the bioengineering of CRISPR/Cas9 and Base-/Prime-editors, the MPI of Biochemistry in Martinsried/Munich on Sendai based Virotherapy, and the biotech company CureVac on mRNA production for gene therapy, allows the development of safe, precise and affordable Gene and RNA therapy and cell therapy products for clinical use.

The existence of a Center for Rare Diseases and several International Registries for Rare Diseases at the Tübingen University working closely with the Institute of Medical Genetics and Applied Genomics, Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE), Comprehensive Cancer Center Tübingen-Stuttgart (CCC-TS), National Center of Tumor Diseases (NCT) as well as the Tübingen Early Clinical Trials Unit (ECTU) enables the identification of novel targets for Gene and RNA therapy and, importantly, allows high-volume recruitment of patients for both academic investigator-initiated (IITs) as well as Pharma trials. This makes Tübingen a highly attractive place for Gene and RNA therapy trials.

Nucleic acids (DNA and RNA) carry the code for life, providing a major platform to regulate health and disease. In fact, the journey of discovery of nucleic acids functions in health and disease started in Tübingen in 1869, when Friedrich Miescher identified a new substance called ‘nuclein’ in the nuclei of immune cells. This discovery ignited a revolution in life sciences and more recently in medicine, offering major innovations in diagnostics and therapies. Gene (DNA) and RNA therapy provides treatment options for disorders traditionally considered ‘undruggable’ and therefore is expected to extend the scale of treatable diseases dramatically, resulting in a medical revolution similar to that of monoclonal antibodies in the past 40 years. Gene and RNA therapy also offers fast-evolving, advanced tools to treat common and rare diseases, showing rapid and groundbreaking progress and revolutionizing medicine already now and in the near future.

Schmuckbild: Biochemische Präparatesammlung des MUT
Biochemical preparation collection in the MUT

Directorate

Prof. Dr. Julia Skokowa, MD, PhD

Founding Director, Gene and RNA Therapy Center
Head, Division of Translational Oncology
Department of Oncology, Hematology, Clinical Immunology and Rheumatology
University Hospital Tübingen

Address: Otfried-Müller-Str. 10
72076 Tübingen

Phone number: +49 7071 29-82168

Fax number: +49 7071 29-25161

E-mail address: Julia.Skokowa@med.uni-tuebingen.de

Scientific coordination

Dr. Olga Klimenkova

Scientific Coordinator, Gene and RNA Therapy Center
Department of Oncology, Hematology, Clinical Immunology and Rheumatology
University Hospital Tübingen

Address: Otfried-Müller-Str. 10
72076 Tübingen

Phone number: +49 7071 29-86013

E-mail address: olga.klimenkova@med.uni-tuebingen.de

Coordinator / Director: Prof. Dr. Julia Skokowa, MD, PhD 

External Advisory Board - Advising the Board of Directors

The board is composed of the spokespersons of the 4 areas:

  • Gene Supplementation Therapy
  • Genome editing
  • Oligonucleotide therapies
  • Virotherapy / Oncolysis

The election of the Board of Directors and the Management Committee is held every 5 years.

Matthew Porteus (Chair), Professor, Department of Pediatrics- Stem Cell Transplantation, Stanford Medical School, USA.

Annemieke Aartsma-Rus, Professor of Translational Genetics, Department of Human Genetics at Leiden University, The Netherlands.

Alberto Auricchio, Associate Professor of Medical Genetics at the University of Naples and Director of Research at the Telethon Institute of Genetics and Medicine in Naples, Italy.

Christopher Baum, Professor, Chief Translational Research Officer / Chairman of the Board of the Berlin Institute of Health, Charite, Berlin, Germany.

Hildegard Bühning, Professor of Infection Biology and Gene Transfer and Deputy Director of the Institute of Experimental Hematology, Hannover Medical School, Germany. President of the European Society for Gene and Cell Therapy and Scientific Secretary of the German Society for Gene Therapy.

Christine Engeland, Professor of Experimental Virology at Witten/Herdecke University, Germany and Head of the Research Group "Mechanisms of Oncolytic Immunotherapy" at Heidelberg University Hospital, National Center for Tumor Diseases and German Cancer Research Center in Heidelberg.

Boris Fehse, Professor, Head of the Cell and Gene Therapy Research Unit at the Clinic for Stem Cell Transplantation, UKE Hamburg. Scientific Advisory Board (past president) of the Society for Gene Therapy.

Anastasia Khvorova, Professor at the RNA Therapeutic Institute, UMass Medical School, USA.

Martin Meier, PhD, Senior Vice President, Research, Alnylam Pharmaceuticals.

Luigi Naldini, Professor of Cell and Tissue Biology and Gene and Cell Therapy at San Raffaele University School of Medicine. Director of the San Raffaele Telethon Institute for Gene Therapy, Milan, Italy.

Dirk Nettelbeck, PD Dr.,Head of the research group Oncolytic Adenoviruses in the Clinical Cooperation Unit Virotherapy at the German Cancer Research Center (DKFZ) in Heidelberg.

Autosomal recessive and early onset ataxias

Friedreich's ataxia

Glioblastoma

Hearing impairment

Hereditary spastic paraplegia

Areas

GRTC at a glance

18
Research Groups
>22
Mio EUR - Funds
136
Employees
54:46
Gender Ratio
22
Patents
263
Publications
46
Doctoral thesis
2
Start-ups
79
Clinical trials
30
Preclinical trials
19
National and international registries / networks
17
Nonprofit patient associations

Clinical Trials

  • Atorvastatin in SPG5 (IIT) (Schöls et al. Brain 2017)
    PI: Dr. Ludger Schöls 
  • ·Cas9-Gen Editing für Sichelzellenanämie und β-Thalassämie. N Engl J Med. 21;384(3):252-260. doi: 10.1056/NEJMoa2031054.
    ClinicalTrials.gov Identifier: NCT03655678
    Link: https://clinicaltrials.gov/ct2/show/NCT03655678
    PI: Dr. Markus Mezger 
  • Eine blinde, placebokontrollierte Phase-1-Studie zur Untersuchung der Sicherheit, Verträglichkeit und Pharmakokinetik von mehreren aufsteigenden Dosen von BIIB132, die Erwachsenen mit spinozerebellärer Ataxie 3 intrathekal verabreicht werden (MERA) (Biogen)
    ClinicalTrials.gov Identifier: NCT05160558
    Link: https://clinicaltrials.gov/ct2/show/NCT05160558
    PI: Dr. Matthis Synofzik 
  • Eine klinische Studie der Phase 1b/2 zur intratumoralen Verabreichung von V937 in Kombination mit Pembrolizumab (MK-3475) bei Teilnehmern mit fortgeschrittenen/metastasierten soliden Tumoren - VIROTHERAPY STUDY
    ClinicalTrials.gov Identifier: NCT04521621
    Link: https://clinicaltrials.gov/ct2/show/NCT04521621
    PI: Dr.Ulrich Lauer 
  • Eine multizentrische, nicht-randomisierte, offene, adaptive Phase-I/II-Studie mit 5-Jahres-Follow-up und Einzeldosis-Eskalation von VTX-801 bei erwachsenen Patienten mit Morbus Wilson"; GENE THERAPY STUDY
    ClinicalTrials.gov Identifier: NCT04537377
    Link: https://clinicaltrials.gov/ct2/show/NCT04537377
    PI: Dr.Ulrich Lauer 
  • Eine multizentrische, offene Phase-1b/2-Studie zur Bewertung der Sicherheit von Talimogene Laherparepvec, dass allein und in Kombination mit systemischem Pembrolizumab in Lebertumore injiziert wird - VIROTHERAPY STUDIE
    ClinicalTrials.gov Identifier: NCT02509507
    Link: https://clinicaltrials.gov/ct2/show/NCT02509507
    PI: Dr.Ulrich Lauer 
  • Eine multizentrische, offene Phase-1b/3-Studie mit Talimogene Laherparepvec in Kombination mit Pembrolizumab (MK-3475) zur Behandlung von nicht resezierten Melanomen im Stadium IIIB bis IVM1c (MASTERKEY-265) - VIROTHERAPY STUDY
    ClinicalTrials.gov Identifier: NCT02263508
    Link: https://clinicaltrials.gov/ct2/show/NCT02263508
    PI: Dr.Ulrich Lauer 
  • Eine Phase 1 / 2-Studie zu Biomarkern, Sicherheit und Wirksamkeit bei fortgeschrittenem oder metastasiertem Magen-Darm-Krebs, die Behandlungskombinationen mit Pelareorep und Atezolizumab untersucht - VIROTHERAPY STUDIE (AIO-KRK-0320/ass.; EudraCT 2020-003996-16)
    PI: Dr.Ulrich Lauer 
  • Eine Phase-1b-Studie von Talimogene Laherparepvec in Kombination mit Atezolizumab bei Patienten mit dreifach negativem Brustkrebs und Darmkrebs mit Lebermetastasen - VIROTHERAPY STUDY
    ClinicalTrials.gov Identifier: NCT03256344
    Link: https://clinicaltrials.gov/ct2/show/NCT03256344
    PI: Dr.Ulrich Lauer 
  • Eine randomisierte, doppelblinde, placebokontrollierte Parallelgruppenstudie zur Bewertung der Wirksamkeit, Sicherheit und Verträglichkeit von BIIB080 bei Patienten mit leichter kognitiver Beeinträchtigung aufgrund der Alzheimer-Krankheit oder leichter Alzheimer-Demenz (CELIA) (Biogen)
    ClinicalTrials.gov Identifier: NCT05399888
    Link: https://clinicaltrials.gov/ct2/show/NCT05399888
    PI: Dr. Matthis Synofzik 
  • Eine randomisierte, offene Phase-3-Studie zum Vergleich von Pexa-Vec (Vaccinia GM-CSF / Thymidine Kinase-deaktiviertes Virus) gefolgt von Sorafenib versus Sorafenib bei Patienten mit fortgeschrittenem hepatozellulärem Karzinom (HCC) ohne vorherige systemische Therapie - VIRO-THERAPY STUDY
    ClinicalTrials.gov Identifier: NCT02562755
    Link: https://clinicaltrials.gov/ct2/show/NCT02562755
    PI: Dr.Ulrich Lauer 
  • Multizentrische, randomisierte, doppelblinde, placebokontrollierte Phase-1b/2a-Studie von WVE-004, intrathekale Verabreichung an Patienten mit C9orf72-assoziierter Amyotropher Lateralsklerose (ALS) oder Frontotemporaler Demenz (FTD) (WAVE)
    ClinicalTrials.gov Identifier: NCT04931862
    Link: https://clinicaltrials.gov/ct2/show/NCT04931862
    PI: Dr. Matthis Synofzik 
  • Offene Phase-I-Dosis-Eskalationsstudie von BI 1831169 als Monotherapie und in Kombination mit Ezabenlimab bei Patienten mit fortgeschrittenen oder metastasierten soliden Tumoren - VIROTHERAPY STUDY
    ClinicalTrials.gov Identifier: NCT05155332
    Link: https://clinicaltrials.gov/ct2/show/NCT05155332
    PI: Dr.Ulrich Lauer (Please link to Members section)
  • Phase-I/II-Studie zur intraperitonealen Verabreichung von GL-ONC1, einem gentechnisch veränderten Vaccinia-Virus, bei Patienten mit Peritonealkarzinose - VIROTHERAPY STUDY
    ClinicalTrials.gov Identifier: NCT01443260
    Link: https://clinicaltrials.gov/ct2/show/NCT01443260
    PI: Dr.Ulrich Lauer 
  • PIGMENT - PDE6A Gentherapie für Retinitis Pigmentosa  
    (Gen Augmentation in PDE6A-arRP. AAV8, subretinal, einmalige Injektion)
     ClinicalTrials.gov Identifier: NCT04611503
    Link: https://clinicaltrials.gov/ct2/show/NCT04611503
    PI: Dr. Bernd Wissinger 
  • Sicherheit und Wirksamkeit einer bilateralen subretinalen Einzelinjektion von rAAV.hCNGA3 bei erwachsenen und minderjährigen Patienten mit CNGA3-verknüpfter Achromatopsie, untersucht in einer randomisierten, Wartelisten-kontrollierten, Beobachter-maskierten Studie (Gen-Augmentation bei CNGA3-ACHM. AAV8, subretinal, einmalige Injektion)
    ClinicalTrials.gov Identifier: NCT02610582
    Link: https://clinicaltrials.gov/ct2/show/NCT02610582
    PI: Dr. Bernd Wissinger
  • Reichel FF et al. Dreijährige Ergebnisse der Phase I der retinalen Gentherapie für CNGA3-mutierte Achromatopsie: Ergebnisse einer nicht randomisierten kontrollierten Studie. Br J Ophthalmol. 2021 May 18:bjophthalmol-2021-319067. 
  • Fischer et al. Sicherheit und Sehleistung einer subretinalen Gentherapie, die auf Zapfenphotorezeptoren bei Achromatopsie abzielt: Eine nicht randomisierte, kontrollierte Studie.JAMA Ophthalmol. 2020 Jun 1;138(6):643-651 
  • Vatiquinone in FA (Phase 3)
    PI: Dr. Ludger Schöls
  • Tofersen (BIIB067) bei Amyotropher Lateralsklerose in Verbindung mit einer Mutation im Superoxid-Dismutase-1-Gen: Compassionate-Use-Programm (Biogen)
    ClinicalTrials.gov Identifier: NCT04856982
    Link: https://clinicaltrials.gov/ct2/show/NCT04856982
    PI: Dr. Matthis Synofzik
  • Erstes humane n-of-1-Behandlungsprogramm maßgeschneiderte n-of-1-ASO-Behandlung für Ataxia Teleangiectasia (zusammen mit Boston Children's Hospital, Harvard)
    PI: Dr. Matthis Synofzik

News

Gene therapy cures deafness

Gene therapy cures a special form of congenital deafness

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Breakthrough Prize Life Sciences

Thomas Gasser, Hertie Institute for Clinical Brain Research ...

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DG-GT Annual meeting 2024

The next Annual Meeting of the German Society for Gene Therapy.

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