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Tuesday 11 October




Education Session

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Clinical Trials and Commercialisation Workshop


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Main Congress Registration

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Pentland Suite

Plenary 1

ESGCT 2022 Opening Keynotes

Chairs: Hildegard Büning, Hannover Medical School / Rafael Yañez-Muñoz, Royal Holloway University of London / Andrew Baker, University of Edinburgh 

14:00 - 14:30: Welcome words

Andy Baker, University of Edinburgh; Rafael Yáñez-Muñoz, Royal Holloway University of London, Hildegard Büning, Hannover Medical School

INV01: Odile Cohen-Haguenauer, Hopital Saint Louis, Paris

30 years of the ESGCT


INV02: Viviana Gradinaru, CalTech

Getting across barriers: Gene delivery across the blood-brain barrier for precise and minimally-invasive study and repair of nervous systems

INV03: Stuart Forbes, University of Edinburgh

Developing cell therapies for liver disease


16:30 - 17:00

Coffee Break


Pentland and Sidlaw

Parallel 1a

Gene & epigenetic editing 

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

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Parallel 1b

Haematopoietic stem cell therapies in immunodeficiencies and metabolic disorders


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

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Lomond Suite

Parallel 1c

In vivo gene therapy & immune responses

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

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Welcome Reception

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