Print Programme

Annual meeting of SFTCG 

ENS, Lyon, France 

11 to 13 March, 2020

Wednesday 11 March

09:00

12:00

Matinée portes ouvertes pour les lycéens de Lyon Speed-searching: Interaction avec les chercheurs et leurs outils de travail

12:30 13:45

Session pour les patients et associations de patients
1) Introduction générale sur la thérapie génique
2) Atelier individuel sur différents thèmes
3) Table Rond introduit par un film
13:30  Registration

14:00
14:45
 

Opening Keynote
Chairs François-Loïc Cosset, Els Verhoeyen
INV01  Nathalie Cartier,  ICM, Paris
Gene therapy for CNS

14:45 16:45

Session 1 : Gene editing in gene therapy
Chairs: François-Loïc Cosset, François Moreau-Gaudry 

INV02 Claudio Mussolino, Freiburg University
Advances in precision genome and epigenome editing for human gene therapy
INV03 Fernando Larcher, Ciemat / Ciberer, Madrid
CRISPR/Cas9-based gene editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa
INV04 Annarita Miccio, Institut IMAGINE, Paris
CRISPR/Cas9-based therapeutic strategies for β-hemoglobinopathies

OR01  Florence Prat, Institut des biothérapies des maladies génétiques, inflammatoires et cancer, Bordeaux
Mutation-specific guide-RNA for compound heterozygous disease scar-less correction by CRISPR-Cas9 in stem cells
OR02  Carla Sanjurjo-Soriano, Inserm U1051, Institute for Neurosciences of Montpellier
Genome editing in patient iPSC efficiently corrects the most prevalent USH2A mutations and reveals pathophysiological mechanisms in iPSC-derived retinal organoids

16:45 

17:15 

Coffee break

17:15

19:15 

Session 2 : In vivo gene therapy
Chairs: Eduard Ayuso, Capucine Trollet

INV05  Gloria Gonzalez- Aseguinolaza,   CIMA, Pamplona
Gene therapy for inherited liver diseases
INV06  Martina Marinello, Genethon, Evry
Gene therapy of spinal muscular atrophy by single-stranded AAV9 vectors
INV07  Els Verhoeyen (On behalf of Fred Thalheimer,  Paul Elrich Institute, Langen)
In vivo
generation of functional CAR T cells selectively in human CD4+ lymphocytes

OR03  Karima Habbas, University of Strasbourg
DgkK as a new therapeutic target in the Fmr1-KO murine model of Fragile X syndrome
OR04 Dries David, KU Leuven
First steps towards a kidney directed gene therapeutic approach for the lysosomal storage disease cystinosis

SFTCG General Assembly

19:15 

Welcome reception and Posters

 
Thursday 12 March

9:00

11:00

Session 3: Innovative approaches
Chairs: Michel Pucéat, Els Verhoeyen

INV11 Tuan Nguyen, Golliver Therapeutics, Nantes 
Pluripotent stem cell-based ATMPs for liver repair
INV12 Jens Schwamborn, University of Luxemburg
Human midbrain organoids for Parkinson`s disease in vitro modeling and drug development 
INV13 Valérie Dardalhon, IGMM, Montpellier 
Targeting the metabolic environment to modulate CAR-T cell effector function

OR05  Aurélien Leray, CEISAM UMR 6230, University of Nantes
Chemically modified AAV vectors for gene therapy
OR06  Lorraine Quillien, Inserm U1037 Equipe 10, Toulouse
First-in-class imaging approach for single-cell, real-time analysis of oncolytic virus replication and efficacy in cancer cells

11:00

11:30

Coffee Break

11:30

13:30

Session 4: Cancer gene therapy
Chairs: Nicolas Boisgerault, Pierre Cordelier

INV14 Andrea Biondi, University of Milano Bicocca, Monza 
Allogeneic CD19 CAR Cytokine Induced Killer (CIK) cells with Sleeping Beauty (SB) transposon for adoptive immunotherapy
INV15 Nicolas Ferry, Servier
CAR-T cells, the slow road from autologous to allogenic origin
INV16 Yaohe Wang, Barts Cancer Institute, London
The potential of a new intravenously deliverable oncolytic vaccinia virus for cancer treatment 

OR07 Els Verhoeyen, CIRI; Inserm U1111, Lyon
Efficient and robust NK-cell transduction with baboon envelope pseudotyped lentivector: A new tool for CAR T cell immunotherapy  
OR08 Alvaro Morales Molina, Instituto de Salud Carlos III, Madrid
Cellular immunotherapy using oncolytic adenoviruses induces antitumor effect and higher infiltration of T cells in mouse models of melanoma, renal cancer and osteosarcoma

13:30

15:00

Lunch and Posters

15:00

17:00

Session 5 : Stem Cells and iPS
Chairs: John De Vos, Leila Maouche-Chrétien

INV08 Guillaume Rousseau, EryPharm, Paris  
Ex vivo production of red blood cells from stem cells for transfusion purposes
INV09 Pierre Savatier, Stem Cell and Brain Research Institute, Lyon
Naive-state pluripotency and chimeric competency in human and non-human primates
INV10 Christelle Monville, Istem, Evry 
Retinal pigment epithelial cells derived from human embryonic stem cells disposed on human amniotic membrane: An update on the french RP clinical trial

OR09  Virginie Lambert, Hôpital Bicêtre, Paris
Cell therapy to treat right heart failure: Benefits of human cardiac progenitors cell-seeded collagen patches on failing right ventricular myocardium

OR10  Victor Tiroille, Université de Nice
UBTD1 is a mechano-regulator controlling stem cell behavior in prostate organoid: Genome editing by nanoblade technology & retroviral strategy

17:00

17:30

Coffee Break

17:30

19:30

Session 6: Towards clinical trials
Chairs: Matthias Titeux, Françoise Piguet

INV17 Françoise Piguet, Inserm, Paris
Restoring neuronal cholesterol metabolism in ALS mouse model
INV18 Matthias Titeux, Institut Imagine, Paris
EBGRAFT clinical trial: autologous gene corrected skin graft for Recessive Dystrophic Epidermolysis Bullosa
INV19 Anne Galy, Genethon Evry
Understanding and managing regulatory and reimbursement uncertainty during ATMP development: commercial and business impacts

OR11 Joffrey Mianné, IRMB, Univ Montpellier, INSERM, CHU Montpellier
iPSCs-derived airway epithelium for primary ciliary dyskinesia modelling and investigation of personalized CRISPR/Cas9 gene therapy
OR12 Anne Catherine Prats, I2MC, Inserm UMR 1048
Towards a clinical assay of combined gene therapy to cure chronic heart failure

20:00 Congress dinner and party
 
Friday 13 March

09:30

11:00

Session 7: Clinical trials & more
Chairs: Olivier Nègre, Juan Bueren
INV20 Christian Jorgensen, IRMB, Montpellier
State of play of regenerative therapy in degenerative disc disease
INV21 Michael Hocquemiller, Lysogene
LYS-SAF302 gene therapy study in mucopolysaccharidosis type IIIA (MPS IIIA) children
 

OR13  Barbara Garmy Susini, Inserm UMR1048, Toulouse
Theralymph : An innovative  H2020 program to perform gene therapy for Lymphedema
OR14 Rebecca Xicluna, Université de Nantes
Characterization of anti-AAV viral pre-existing cellular immune responses in humans suggests a potential key role for TEMRA CD8+T cells

11:00

11:30

Coffee break

11:30

12:30

Closing keynote
Chairs: Els Verhoeyen

INV23  Juan Bueren, CIEMAT/Ciberer, Madrid
Gene therapy in hematopoietic stem cell diseases: The Fanconi anemia model

Poster and oral presentation awards