PROGRAMME
Wednesday 19 March
09:30-12:30
Matinée d'information pour les lycéens
12:00-13:00
Registration
No lunch provided so you need to make sure you bring something or you eat before you arrive as there is not much around
13.00-14.00
Opening keynote
Chairs: François Moreau Gaudry, Oumeya Adjali
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13:00 Welcome
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13:15 Gregory Newby, John Hopkins University
INV01 Precision genome editing to treat genetic disease
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14.00-16.00
Session 1: Advances and challenges for gene editing technology
Chairs: François Moreau Gaudry, Oumeya Adjali
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​14:00 Anna Cereseto, University of Trento
INV02: Expanding the genome editing toolbox through the identification and evolution of novel RNA guided nucleases.
14:30 Aurélie Bedel, University of Bordeaux, INSERM U1312
INV03: Challenges for CRISPR-Cas9 genotoxicity: detection and prevention
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15:00 Jose Roberto Alvarez-Vargas, Museum National d'Histoire Naturelle ; Inserm U1154 ; CNRS UMR7196
OR01: POLYPRIME: A novel DNA-Polymerase based prime editing strategy
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15:15 Chloé Thibault, BRIC-BoRdeaux Institute of oncology, Inserm / University of Bordeaux
OR02: Zip Editing: an easy-to-use tool to increase CRISPR-Cas9 HDR-editing efficiency
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15:30 Sébastien Viel, Hospices Civils de Lyon
OR03: Correcting Autoinflammation in STING-Associated Vasculopathy with Onset in Infancy (SAVI) by Human Stem Cell Genome-Editing
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15:45 Paola Galbiati, Genethon, UMR_S951, Inserm, Univ Evry, Université Paris Saclay, EPHE
OR04: CRISPR activation of utrophin as a mutation-independent approach for Duchenne Muscular Dystrophy therapy
16:00- 16:30
Coffee break
16:30-17:00
Poster pitches
17.00-19.00
Session 2: Preclinical and clinical gene editing for gene therapy
Chairs: Els Verhoeyen, Aurélie Bedel
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17:00 Matthew Porteus, Stanford University
INV04: Genome Editing of Stem Cells: Applications to Genetic and Non-Genetic Diseases.
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​17:30 Mario Amendola, Genethon, Evry​
INV05: Engineering Hematopoietic stem cells by targeted gene addition
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​18:00 Paula Rio, Ciemat/Ciberer, Madrid​
INV06: Advancing Hematopoietic Stem Cell Gene Therapy in Fanconi Anemia
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18:30 Marie Hautbois, Laboratory of Genetic skin diseases, INSERM UMR 1163, Imagine Institute, Paris
OR05: Efficient in vitro correction of a highly recurrent COL7A1 pathogenic variant using Cytosine Base editing to treat recessive dystrophic epidermolysis bullosa
18:45 Maëlle Ralu, Genethon, UMR_S951, Inserm, Univ Evry, Université Paris Saclay, EPHE
OR06: CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy
20:00
Party at Mama Shelter
Thursday 20 March
08:30-09:00
Registration
09.00-10.45
Session 3: AAV and lentiviral vectors for gene therapy
Chairs: Caroline Le Guiner, Françoise Piguet
09:00 Alessio Cantore, SR-Tiget, Milano
INV07: Deliver to the liver: towards one-and-done gene therapies for defects of hepatic metabolism
09:30 Hildegard Büning, Hannover Medical School
INV08: Tailored biological nanoparticles for in vivo gene therapy
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10:00 Héloïse Delépée, TaRGeT, Inserm, UMR 1089, Nantes University
OR07: Bioconjugation of the capsid of adeno-associated viruses for osteoarthritis treatment by gene therapy
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10:15 Marion Derome, Genethon, UMR_S951, Inserm, Univ Evry, Université Paris Saclay, EPHE
OR08: AAV-mediated gene therapy of acid ceramidase deficiency by intravenous and CSF-directed vector administration
10:30 Jean Sébastien Diana, Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique-Hôpitaux de Paris, INSERM,
OR09: Gene therapy for familial hemophagocytic lymphohistiocytosis related to Munc 13-4 deficiency
10:45-11:15
Coffee break
11.15-12.45
Session 4: Cancer Therapy I - Immunotherapy and CAR-T cells
Chairs: Nicolas Boisgérault, Mario Squadrito
11:15 Naomi Taylor, Université de Montpellier, NIH
INV09: Harnessing TCR-controlled fuzzy logic to achieve immunotherapeutic precision of CAR T-cells
11:45 Els Verhoeyen, University of Nice
INV10: T-cell targeted lentiviral pseudotypes for in vivo CAR T cell cancer therapy
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12:15 Chiara Martinello, C3M, Université Côte d’Azur, INSERM U1065, Nice
OR10: In vivo CAR T cell therapy against angioimmunoblastic T cell lymphoma
12:30 Tifanie Blein, INSERM UMR 1163, Institut Imagine, Paris Descartes University-Sorbonne Paris Cité
OR11: Combination of TCR-deficient CAR-Tregs and non-mitogenic antiCD3 to promote transplant tolerance.​
12:45-14:00
Lunch and poster session
14.00-16.15
Session 5: Disease model : IPSc and organoids cell therapy
Chairs: Christelle Monvile, Emmanuel Payen
14:00 Olivier Goureau, Institut de la Vision, Paris
INV11: Retinal organoids for modeling inherited retinal diseases and developing new therapeutic strategies
14:30 Elisa Zuccoli, University of Luxemburg
INV12: Patient specific midbrain organoids and assembloids for in vitro disease modeling
15:00 Saverio Tedesco, UCL London​
INV13: Engineering human skeletal muscle for advanced modelling of neuromuscular diseases and gene therapies.
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15:30 Nopmullee Tanhuad, UMR1184 CEA INSERM
OR12: A brain organoid model to study gene and cell therapy treatment for neuronopathic Gaucher disease
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15:45 Clément Morival, Université de Nantes
OR13: Modelling Stargardt disease using three-dimensional retinal organoids
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16:00 Agathe Coeur, IRMB, University of Montpellier, INSERM U1183, CHU Montpellier, Montpellier
OR14: Design of experiments assisted optimization of induced pluripotent stem cell (iPSC) directed differentiation toward airway progenitors for a cell and gene therapy against primary ciliary dyskinesia.
16:15-16:45
Coffee break
16.45-18.15
Session 6: Gene therapy for genetic diseases
Chairs: Gwladys Gernoux, Mario Amendola
16:45 Françoise Piguet, ICM, Paris
INV14: Gene therapy of lysosomal storage disorders
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17:15 Matthias Titeux, Institut Imagine, Paris
INV15: Splice modulation strategies using antisense oligonucleotides for Dystrophic Epidermolysis Bullosa
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17:45 Ji Gao-Desliens, Genethon, Evry
OR15: 3-year Follow-up of Crigler-Najjar Syndrome Patients Treated with AAV-based Gene Therapy in the GNT-012-CRIG Study
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​18:00 Boris Bessot, Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, AssistancePublique-Hôpitaux de Paris, INSERM
OR16: Gene therapy for Artemis-SCID patients: preliminary results of the French ARTEGENE phase I/II clinical trial
18:15-20:00
Poster session 2 and cocktail reception
20:00
Mini night time city tour, dessert and drinks
Only for those who have registered to this event. Limited places available
Friday 21 March
08:00-08:30
Registration
08.30-10.00
Session 7: Cancer therapy II
Chairs: Naomi Taylor, Olivier Nègre
08:30 Mario Squadrito, SR-Tiget, Milan
INV16: In vivo liver macrophage engineering for cancer immunotherapy
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09:00 Laurie Menger, Institut Gustave Roussy
INV17: In vivo genome-wide CRISPR screens for advanced T cell therapy
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09:30 Guénolé Tossou, INSERM UMR1342
OR17: Enhancing anti-tumoral properties of specific extracellular vesicles via miRNA loading: A novel approach for gene therapy in breast and pancreatic cancer models
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09:45 Julia Rossi, Team Biotherapies Genetics and Oncology, BoRdeaux Institute of onCology (BRIC) U1312 University of Bordeaux
OR18: Lentivector onco-targeting for solid tumor gene therapy
10:00-10:30
Coffee break
10.30-12.00
Session 8: Innovative non viral and viral technologies
Chairs: Sophie Gomez, Olivier Goureau
10:30 Jacob Giehm Mikkelsen, Aarhus University
INV18: Engineered lentivirus-derived particles for CRISPR RNP delivery and prime editing
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11:00 Thérèse Cronin, TaRGet - Translational Research in Gene Therapy- INSERM UMR 1089 - IRS 2 Nantes Biotech – Nantes University
INV19: Overcoming limited cargo capacity of AAV vectors in gene editing
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11:30 Charleen Plaisse, Nantes Université, CHU Nantes, INSERM, Center for Research in Transplantation and Translational Immunology, UMR 1064
OR19: Universal allogeneic regulatory T cell therapy from genetically engineered iPSCs
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11h45 Nicolas Ayas, INSERM UMR 1163, Institut Imagine, Université Paris Cité
OR20: Treg empowering through gene addition
12:00-13:15
Lunch and posters
13.15-15.00
Session 9: Innovative approaches
Chairs: Capucine Trollet, Matthias Titeux
13:15 Jean-Luc Perfettini, Gustave Roussy, Paris
INV20: Next-generation human chimeric antigen receptor monocytes for cancer immunotherapy.
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13:45 Guy Lenaers, Université d'Angers
INV21: RNA trans-splicing based gene therapy: a relevant approach to treat diseases related to complex genes.
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14:15 Clémence Lièvre, Inserm UMR 1089
OR21: Skeletal muscle organoids for preclinical gene therapy with recombinant AAV vectors
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14:30 Marie Dewannieux, INSERM US35 - ART-TG
OR22: Viral restriction factors impede the transduction efficiency of gene therapy lentiviral vectors and may be blocked to achieve therapeutic levels of gene transfer
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14:45 Lindsay Jeanpierre, Genethon, UMR_S951, Inserm, Univ Evry, Université Paris Saclay, EPHE
OR23: Muscle-specific expression reduces early antigen presentation and promotes CD8 T cell tolerance after rAAV gene transfer.
15.00-15.45
Session 10: Closing keynote
Chairs: Oumeya Adjali, François Moreau Gaudry
14:45 Alessandro Aiuti, SR-Tiget, Milan
INV22: Hematopoietic stem cell gene therapy for genetic diseases
15:45 - 16:00
Poster and oral presentation awards