Friday 13 March 2020


Public day
for schools and general public


Monday 16 March 2020




Educational session
Chairs: Boris Fehse, University of Hamburg - 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, University of Hamburg
Evolving molecular scissors for antiviral gene therapy
INV05- David Williams, Harvard Medical School
Bringing gene therapy to the clinic and the market

12:00-13:30 LUNCH


Boris Fehse, Axel Schambach, DG-GT
Michael Manns, MHH
Pavel Dutow, Volkswagen Stifftung 

Session I: Delivery
: Hildegard Büning, Axel Schambach
Short introduction
INV06- Els Verhoeyen, Université de Nice
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 
Stabilized non-immunogenic messenger RNA (SNIM® RNA) for transcript therapy

OR01 - 
OR02 - 

16:15-16:45 COFFEE BREAK

Session II: Optimizing the cargo and the microenvironment

Short introduction
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, Hannover Medical School
In vitro models of the hematopoietic stem cell niche: from fundamental research to application as test systems.

OR03 - 
OR04 - 

19:00-19:15 COMFORT BREAK


Key Note 1 – Wolfram Ostertag Lecture
Christopher Baum
INV12: David Williams, Harvard Medical School`
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
Short introduction
INV13- Ulrich Martin, Hannover Medical School
Title TBC
INV14 - Hans Schöler, MPI Münster
Pluripotent Stem Cells and Organoids in Regenerative Medicine and Drug Screening?
INV15 - Bruce Trapnell, Cincinnati Hospital Medical Center
Title TBC

OR05 - 
OR06 - 

11:15-11:45 COFFEE BREAK

Session IV: Evolving topics in gene and cell therapy I
Short Introduction
INV16 - Nico Lachmann, Hannover Medical School
Using the plasticity of macrophages to establish cellular therapies for rare and common diseases
INV17- Joachim Hauber, HPI Hamburg
HIVCURE – A first-in-human phase Ib/IIa gene therapy trial directly targeting HIV-1

13:00-14:00 LUNCH

Keynote 2

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 II
INV19- Hinrich Staecker, University of Kansas
Title TBC

OR07 - 

15:30-16:00 COFFEE BREAK

Session V: Novel approaches in cancer therapy
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 - 
OR09 - 

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
Short introduction
INV24- Toni Cathomen, University of Freiburg
On- and off-target effects of gene editing tools in human stem cells
INV25- Virgis Siksnys, Institute of Biotechnology Vilnius University
Novel CRISPR-Cas nucleases as potential genome editing tools?
INV26- Frank Buchholz, Technische Universität Dresden
Genome Editing with Designer-Recombinases

OR10 - 
OR11 - 

11:15-13:00 POSTER SESSION and LUNCH

Session VII: Clinical translation of gene and cell therapies
Short introduction
INV27- Adrian Thrasher; University College London
Title TBC
INV28- Thomas Thum, Hannover Medical School
(Pre)clinical development of noncoding RNA based therapeutics?
INV29-Fatima Bosch, UAB, Barcelona
Title TBC

OR13 - 
OR14 - 

15:15- 15:45 COFFEE BREAK

Session VIII: Ethical considerations and challenges for the future
Short introduction
INV30- Tobias Cantz, Hannover Medical School  & Thomas Heinemann,
Emerging bioethical challenges in gene therapy approaches
INV31- Nathalie Cartier, Inserm, Paris
Title TBC

Closing & Awards, Hildegard Büning