Tuesday 24 October
08:30-09:00
Registration
09:00-12:45
Education Session
More Information soon >
09:00-12:45
Clinical Trials and Commercialisation Workshop
More Information soon >
13:00-14:00
Main Congress Registration
14:00-16:30
Plenary 1
ESGCT 2023 Opening Keynotes
Chairs: Juan Bueren, Ciemat, Madrid / Thierry VandenDriessche, VUB, Brussels / Els Verhoeyen, University of Nice / Victor Van Beusechem, University of Amsterdam
14:00 - 14:30: Welcome words
Thierry VandenDriessche, Els Verhoeyen, Victor Van Beusechem, Juan Bueren
INV01: Michel Sadelain, Memorial Sloane Kettering Cancer Center, NYC
INV02: Margareth Ozelo, University of Campinas, Hemocentro UNICAMP, Sao Paulo
INV03: TBC
INV04: TBC
16:30 - 17:00
Coffee Break
17:00-19:00
Parallel 2a
Gene editing: Technology development
Chairs: Julian Grunewald, TUM, Munich / Alessia Cavazza, UCL London
INV08: Miguel Esteban, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Science
Identification of mammalian progenitor cells at tissue and body scale
INV09: Angelo Lombardo, SR Tiget, Milan
Exploiting targeted epigenome editing for therapeutic applications
OR10: Rajeev Rai, UCL London
CRISPR/Cas9 based disease modelling and functional correction of Interleukin 7 Receptor Severe Combined Immunodeficiency
OR11: Eleonora Pedrazzoli, University of Trento
CoCas9: a compact nuclease from the human microbiome for efficient and precise editing
OR12: Lucrezia della Volpe, SR Tiget, Milan
Inhibition of p38-MAPK counteracts DNA damage induced by ex vivo expansion of hematopoietic stem and progenitor cells for efficient genetic engineering
OR13: Julia Klermund, University of Freiburg
An in vivo CAST-Seq workflow identifies and quantifies off-target activity as well as chromosomal translocations in organs edited in vivo with CRISPR-Cas nucleases or nickases
P425: Maria Silvia Roman Azcona, University of Freiburg
Contemporary modulation of two major immune checkpoints expression in CAR T cells via hit-and-run epigenome editing
P371: Sébastien Levesque, Université Laval Quebec
MTOR as a selectable genomic harbor for CRISPR-engineered CAR-T cell therapy
17:00-19:00
Parallel 2c
Lentiviral & integrative vectors
Chairs: Gloria Gonzalez-Aseguinolaza, CIMA, Pamplona / Ivana Barbaric, University of Sheffield
INV06: Eric Kremer, University of Montpellier
Why are some adenovirus vectors poorly immunogenic?
INV07: HIldegund Ertl, The Wistar Institute, Philadelphia
Immunotoxicity of high dose AAV vector gene transfer: can we develop protocols that allow for repeated dosing of lower and less toxic doses?
OR06: Greg Gojanovich, AskBio
Using a systems biology approach to unravel the Immunogenicity of AAV8 empty capsids in healthy volunteers.
OR07: Kei Kishimoto, Selecta BioSciences
ImmTOR tolerogenic nanoparticles combined with Treg-selective IL-2 mutein induces massive expansion of antigen-specific regulatory T cells and synergistically inhibit formation of anti-AAV antibodies to high vector doses
OR08: Wolfgang Miesbach, University Hospital Frankfurt
Sustained factor IX activity levels and bleeding protection following etranacogene dezaparvovec administration in people with haemophilia B without and with adeno-associated virus serotype 5 neutralizing antibodies
OR09: Helena Costa Verdera, SR Tiget, Milan
Investigating the role of innate immune signaling in cell toxicity upon AAV-mediated gene transfer in hiPSC-derived CNS models
P423: Ana Cavallo, AstraZeneca
FaDe-Cas9: a fast degrading SpCas9 with reduced cellular immunity
17:00-19:00
Parallel 2b
Lentiviral & integrative vectors
Chairs: Claire Booth, UCL London / Anne Galy, Genethon, Evry
INV04: Frank Staal, Leiden University Medical Center
Developing stem cell based gene therapy for RAG1 and RAG2 deficient SCID.
INV05: Rosa Bacchetta, Stanford School of Medicine
Current Advances in the Gene therapy for IPEX syndrome
OR01: Francesca Ferrua, SR Tiget, Milan
Safety and clinical benefit of lentiviral haematopoietic stem and progenitor cell gene therapy in 23 patients with Wiskott-Aldrich Syndrome with up to 10.5 years of follow-up
OR02: Sameer Bahal, UCL, London
Experience of genome editing patient haematopoietic stem cells to treat X-linked Agammaglobulinemia
OR03: Stuart Ellison, University of Manchester
Enhanced transduction and immunophenotyping demonstrates preclinical safety and efficacy of haematopoietic stem cell gene therapy for Mucopolysaccharidosis II (MPSII) using an IDS.ApoEII brain targeted therapy
OR04: Valentina Poletti, University of Padova
Preclinical development of an ex vivo gene therapy for Mucopolysaccharidosis type II
OR05: Ludovica Santi, SR Tiget, Milan
Skeletal damage and cross-correction in MPSIH HSPC-gene therapy
17:00-19:00
Parallel 2c
Lentiviral & integrative vectors
Chairs: Gloria Gonzalez-Aseguinolaza, CIMA, Pamplona / Ivana Barbaric, University of Sheffield
INV06: Eric Kremer, University of Montpellier
Why are some adenovirus vectors poorly immunogenic?
INV07: HIldegund Ertl, The Wistar Institute, Philadelphia
Immunotoxicity of high dose AAV vector gene transfer: can we develop protocols that allow for repeated dosing of lower and less toxic doses?
OR06: Greg Gojanovich, AskBio
Using a systems biology approach to unravel the Immunogenicity of AAV8 empty capsids in healthy volunteers.
OR07: Kei Kishimoto, Selecta BioSciences
ImmTOR tolerogenic nanoparticles combined with Treg-selective IL-2 mutein induces massive expansion of antigen-specific regulatory T cells and synergistically inhibit formation of anti-AAV antibodies to high vector doses
OR08: Wolfgang Miesbach, University Hospital Frankfurt
Sustained factor IX activity levels and bleeding protection following etranacogene dezaparvovec administration in people with haemophilia B without and with adeno-associated virus serotype 5 neutralizing antibodies
OR09: Helena Costa Verdera, SR Tiget, Milan
Investigating the role of innate immune signaling in cell toxicity upon AAV-mediated gene transfer in hiPSC-derived CNS models
P423: Ana Cavallo, AstraZeneca
FaDe-Cas9: a fast degrading SpCas9 with reduced cellular immunity