Wednesday 16 March

13:00-14:00

Registration

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14:00-14:45

Opening Keynote

Chair: Els Verhoeyen

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INV01  Nathalie Cartier, BrainVectis, AskBio, Paris  
Gene therapy for CNS

14:45-16:15

Session 1: Gene editing in gene therapy

Chairs: François-Loïc Cosset, François Moreau-Gaudry

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INV02  Fernando Larcher, Ciemat / Ciberer, Madrid  
CRISPR/Cas9-based gene editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa


INV03  Annarita Miccio, Institut IMAGINE, Paris  
CRISPR/Cas9-based therapeutic strategies for β-hemoglobinopathies


OR01  Victor Tiroille, Université Côte d’Azur, INSERM, C3M, 06204 Nice, France

Nanoblades allow high-level genome editing in organoids


OR02  Juliette Rosier, Université Bordeaux

CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells leading to imprinting defects

16:15-16:45

Coffee break

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16:45-18:45

Session 2: In vivo gene therapy

Chairs: Eduard Ayuso, Capucine Trollet

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INV04  Gloria Gonzalez-Aseguinolaza, CIMA, Pamplona  
Gene therapy for liver inherited diseases


INV05  Martina Marinello, Genethon, Evry  
Gene therapy of spinal muscular atrophy by single-stranded AAV9 vectors


INV06  Frederic Thalheimer, Paul-Ehrlich-Institut, Langen  
Receptor targeted viral vectors for In vivo delivery


OR03  Celia Sourd, Genethon, UMR_S951, Inserm, Univ Evry, Université Paris Saclay, EPHE

An AAVpo1A1 vector expressing MTM1 corrects skeletal muscle pathology with detargeted liver transduction in myotubular myopathy mice


OR04  Mathieu Mével, Nantes Université, TaRGeT - Translational Research in Gene Therapy, INSERM UMR 1089, CHU de Nantes

Chemically tyrosine modified AAV vectors: an innovative technological platform to boost gene delivery

18:45-19:30

SFTCG General Assembly

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19:15

Welcome reception and Posters

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Thursday 17 March

08:30-09:00

Registration

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09:00-11:00

Session 3: Innovative approaches

Chairs: Michel Pucéat, Els Verhoeyen

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INV07  Denis Furling, Centre de Recherche en Myologie, UMRS 974, Paris  
Decoy gene therapy for Myotonic Dystrophy.


INV08  Eduard Ayuso, Dinaqor, Zurich  

A genetic medicine platform for the treatment of severe inherited cardiac diseases


INV09 Valérie Dardalhon, IGMM, Montpellier  
Targeting the metabolic environment to modulate CAR-T cell effector function


OR05  Audrey Page, CIRI; Inserm U1111

A new synthetic circuit for B cell reprogramming to cure cancer


OR06  Tina Briolay, Université de Nantes

Development of a bio-inspired nanovector for targeted cancer gene therapy

11:00-11:30

Coffee break

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11:30-13:30

Session 4: Cancer gene therapy

Chairs: Nicolas Boisgerault, Pierre Cordelier

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INV10 Axel Schambach, Hannover Medical School 

From CARs to TRUCKs: Towards next-generation immunotherapeutics


INV11  Hélène Nègre, Servier  
T2EVOLVE: Standardization of preclinical and clinical development of engineered T-cell therapy in Europe—a cross-functional multi-stakeholder initiative 


INV12  Els Verhoeyen, CIRI, ENS de Lyon and C3M, Nice      Alejandra Gutierrez-Guerrero, CIRI, ENS de Lyon  
Novel lentiviral pseudotypes for T and NK cell gene therapy and ‘nanoblades’ for efficient gene editing in hematopoietic gene therapy target cells.


OR07  Adrien Krug, Université Côte d'Azur/C3M

CD8-targeted lentiviral vectors to generate CAR T cells in vivo as treatment for T cell lymphoma


OR08  Ugo Hirigoyen, Nantes Université, Inserm UMR 1307, CNRS UMR 6075, Université d'Angers, CRCI2NA

Characterization of tumor extracellular vesicles produced during oncolytic infection

13:30-15:00

Lunch and Posters

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15:00-16:45

Session 5 : Stem Cells and iPS

Chairs: John De Vos, Leila Maouche-Chrétien

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INV13  Guillaume Rousseau, EryPharm, Paris  
Industrial production of cultured red blood cells: a model for future large-scale cell therapies finally within reach


INV14  Pierre Savatier, Stem Cell and Brain Research Institute, Lyon  
Interspecies systemic chimeras : a new path for exploring pluripotent stem cell biology and modeling human early development


INV15  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  Carine Bourdais, IRMB, Univ Montpellier, INSERM, CHU Montpellier

Combined cellular and gene therapy to treat primary ciliary dyskinesia

16:45-17:15

Coffee break

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17:15-19:15

Session 6: Towards clinical trials

Chairs: Anne Galy, Françoise Piguet

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INV16  Françoise Piguet, Inserm, Paris  
Gene therapy of Amyotrophic Lateral Sclerosis


INV17  Olivier Nègre, GCTi  

Gene and cell therapies: G&CTI, a think tank to face the multiple challenges


INV18  Thomas Roujeau, Hôtpital Gui de Chauliac, Montpellier  
Intracerebral gene therapy in 2 patients with aromatic-L acid decarboxylase  (AADC) deficiency


OR11  Miryam Mebarki, Unité de Thérapie Cellulaire, Hôpital Saint-Louis, AP-HP

Development of a human umbilical cord-derived mesenchymal stromal cell (UC-MSC)-based advanced therapy medicinal product (ATMP) to treat immune and/or inflammatory diseases


OR12  Barbara Garmy-Susini, Inserm UMR1048

15-Lipoxygenase drives inflammation resolution and Treg trafficking in lymphedema

 

Friday 18 March

09:00-09:30

Registration

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09:30-11:30

Session 7: Gene therapy & more

Chairs: Olivier Nègre, Juan Bueren

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INV19  Anne Galy, Genethon, Evry  

Gene therapy using lentiviral vectors : current clinical results and novel perspectives


OR13  Elena-Gaia Banchi, Institut du Cerveau et de la Moelle épiniere

Gene Therapy For Spinocerebellar Ataxia 7 : Restoring Cholesterol Metabolism


OR14  Juliette Lemoine, Genethon, UMR_S951, Inserm, Univ Evry, Université Paris Saclay, EPHE

Novel AAV capsid variant for muscle-directed gene therapy 

10:30-11:00

Coffee break

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11:00-12:00

Closing keynote

Chairs: Els Verhoeyen

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INV21  Juan Bueren, CIEMAT/Ciberer, Madrid  
Gene therapy in hematopoietic stem cell diseases: The Fanconi anemia model


Poster and oral presentation awards