Friday 13 March 2020

9.00 – 12.30 

Venue: MHH Lecture hall R

PUBLIC DAY - for School Children (High School/Gymnasium) and general public

Monday 16 March 2020 - Education Session


Venue: Herrenhausen Palace, Lecture hall

Educational session (for PhD Students, MD Students and MD PhD Students)
Chairs: Boris Fehse, University Medical Center Hamburg Eppendorf  Axel Schambach, Hannover Medical School - Hildegard Büning, Hannover Medical School
Welcome words
INV01- Hildegard Büning, Hannover Medical School
Optimizing gene vectors for gene therapy (AAV/LV/Nonviral)
INV02- Axel Schambach, Hannover Medical School
Exploiting stem cells for combined gene and cell therapies
INV03- Toni Cathomen, University of Freiburg
Designer nucleases: Basics, challenges and beyond

10:40-11:00 COFFEE BREAK

INV04- Joachim Hauber, Heinrich-Pette-Institute Hamburg
Evolving molecular scissors for antiviral gene therapy
INV05- David Williams, Harvard Medical School, Cambridge
Bringing gene therapy to the clinic and the market

12:00-13:30 LUNCH

Monday 16 March 2020 - Congress


Boris Fehse, DG-GT
Axel Schambach, LOC
Michael Manns, Hannover Medical School
Pavel Dutow, Volkswagen Foundation

Session I: Delivery
: Hildegard Büning, Axel Schambach
Short introduction in the topics by the chairs
INV06- Els Verhoeyen, University of Nice, ENS, Lyon
Novel lentiviral pseudotypes for T and NK cell gene therapy and ‘nanoblades’ for efficient gene editing in hematopoietic gene therapy target cells.
INV07 - Leszek Lisowski, University of Sydney
Canonical AAV gene therapy and clinical gene editing: Capsid evolution and translational vectorology
INV08 - Carsten Rudolph, Technical University Munich
Stabilized non-immunogenic messenger RNA (SNIM® RNA) for transcript therapy

OR01 - Sabrina Just, Hannover Medical School
Novel AAV capsid variant for muscle-directed gene therapy
OR02 - Fritz Nolte, University Medical Center Hamburg Eppendorf  
Membrane protein-specific nanobodies dramatically increase the transduction efficiencies of AAV vectors

16:15-16:45 COFFEE BREAK

Session II: Optimising the cargo and the microenvironment
: Christian Kupatt and Michael Ott
Short introduction in the topics by the chairs
INV09 - Thierry VandenDriessche, Vrije Universiteit Brussel
Exploiting synthetic biology for gene therapy
INV10 - Alberto Auricchio, TIGEM, Naples
Expanding AAV transfer capacity on the retina
INV11 - Cornelia Lee-Thedieck, Leibniz University Hannover
In vitro models of the hematopoietic stem cell niche: from fundamental research to application as test systems.

OR03 - Marko Radukic, Bielefeld University
Adeno-Associated Virus Transgene Characterization by Direct Nanopore Sequencing
OR04 - Yulia Yuzefovych, Hannover Medical School
Organ gene therapy: Genetic engineering of the kidney towards immunological invisibility

19:00-19:15 COMFORT BREAK


Key Note 1 – Wolfram Ostertag Lecture
Christopher Baum
Short introduction of the award and the awardee by the chair?
INV12: David Williams, Harvard Medical School, Cambridge
Flipping the switch; Post-transcriptional genetic silencing of BCL11A to treat Sickle Cell Disease



Tuesday 17 March 2020


Session III: iPSC- and stem cell-based cell therapies
Chairs: Thomas Moritz, Tobias Cantz
Short introduction in the topics by the chairs
INV13- Ulrich Martin, Hannover Medical School
hiPS cells for heart repair: Generation of GMP-compliant cell lines, large scale cardiomyocyte production production and preclinical evaluation in a non-human primate model
INV14 - Hans Schöler, MPI Münster
Pluripotent stem cells and organoids in regenerative medicine and drug screening
INV15 - Bruce Trapnell, Cincinnati Hospital Medical Center
Pulmonary macrophage transplantation therapy of hereditary PAP: From disease discovery to novel therapy

OR05 - Francesca Fumagalli, SR Tiget, Milan
Lentiviral haematopoietic stem and progenitor cell gene therapy for metachromatic leukodystrophy: Clinical outcomes from 33 patients 
OR06 - Mania Ackermann, Hannover Medical School
Adoptive transfer of human iPSC-macrophages as a cellular therapy to treat gram positive and negative bacterial airway infections

11:15-11:45 COFFEE BREAK

Session IV: Evolving topics in gene and cell therapy
Chairs: Boris Fehse & Christina Falk
Short introduction in the topics by the chairs
INV16 - Nico Lachmann, Hannover Medical School
Using the plasticity of macrophages to establish cellular therapies for rare and common diseases
INV17- Joachim Hauber, Heinrich-Pette-Institute Hamburg
HIVCURE – A first-in-human phase Ib/IIa gene therapy trial directly targeting HIV-1

13:00-14:00 LUNCH and General Assembly

Keynote 2
Brigitte Schlegelberger, Ute Modlich
INV18 - Hans-Peter Kiem, Fred Hutchinson Cancer Research Center, Seattle
Hematopoietic stem cell gene therapy: advances, challenges and opportunities in the new decade


Session IV: Evolving topics in gene and cell therapy
INV19- Hinrich Staecker, University of Kansas
Vectorization approaches for different human inner ear diseases: Regeneration, protection and treatment of genetic disorders

OR07 - Antje Prasse, Hannover Medical School 
Airway basal cells of patients with idiopathic pulmonary fibrosis exhibit increased stemness and promote fibrosis. 

15:30-16:00 COFFEE BREAK

Session V: Novel approaches in cancer therapy
Chairs: Dirk Nettelbeck, Wolfgang Uckert
Short introduction in the topics by the chairs
INV20- Guy Ungerechts, NCT Heidelberg
Immunovirotherapy for Cancer
INV21- Dorothee von Laer, Innsbruck University
Oncolytic rhabdoviruses in cancer immunotherapy
INV22- Martin Sauer, Hannover Medical School
Off the shelf cellular immunotherapy: Dream or Necessity

OR08 - Laura Mosti, University of Freiburg
Generation of safe CAR T cells to target B cell malignancies
OR09 - Florian Kühnel, Hannover Medical School
Molecular retargeting of antiviral antibodies against tumors using bispecific adapter proteins

18:15-18:30 COMFORT BREAK
19:00 - 20:30

Public Lecture 
Axel Schambach & Hildegard Büning
Mit Gentaxis in die Zelle: Auf dem Weg zu neuen Behandlungsstrategien für angeborene und erworbene Erkrankungen

Wednesday 18 March 2020


Session VI: Genome and epigenome editing: On the road towards the clinic
Chairs: Claudio Mussolino, Adrian Schwarzer
Short introduction in the topics by the chairs
INV23- Toni Cathomen, University of Freiburg
On- and off-target effects of gene editing tools in human stem cells
INV24- Virgis Siksnys, Institute of Biotechnology Vilnius University
Novel CRISPR-Cas nucleases as potential genome editing tools?
INV25- Frank Buchholz, Technical University Dresden
Genome editing with designer-recombinases

OR10 - Christian Kupatt, Technical University Munich 
Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy
OR11 - Dawid Glow, University Medical Center Hamburg Eppendorf  
LATE -novel, sensitive cell-based assay for the identification of CRISPR-Cas9 related long-term adverse treatment effects 

11:15-13:00 POSTER SESSION and LUNCH

Session VII: Clinical translation of gene and cell therapies
Chairs: Stefan Kochanek, Ulrike Köhl
Short introduction in the topics by the chairs
INV26- Adrian Thrasher; University College London
Stem cell gene therapy: learning from immunodeficiency and metabolic disease
INV27- Thomas Thum, Hannover Medical School
(Pre)clinical development of noncoding RNA based therapeutics
INV28-Fatima Bosch, UAB, Barcelona
Gene therapy in type 2 diabetes: where are we?

OR12 - Adrian Schwarzer, Hannover Medical School
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning
OR13 - Andre Müller-York, Sarepta Therapeutic inc
Systemic gene transfer with rAAVrh74.MHCK7.SGCB increased sarcoglycan expression in patients with limb girdle muscular dystrophy type 2E 

15:15- 15:45 COFFEE BREAK

Session VIII: Ethical considerations and challenges for the future
Chairs: Toni Cathomen, Zoltan Ivics
Short introduction in the topics by the chairs
INV29- Tobias Cantz, Hannover Medical School  & Thomas Heinemann, Hochschule Vallendar (PTHV) 
Emerging bioethical challenges in gene therapy approaches
INV30- Nathalie Cartier, Inserm, Paris
Gene Therapy for neurodegenerative diseases from genetic to complex diseases : delivery and targets

Closing & Awards, Hildegard Büning