Wednesday 16 March
13:00-14:00
Registration
14:00-14:45
Opening Keynote
Chair: Els Verhoeyen
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
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
16:45-18:45
Session 2: In vivo gene therapy
Chairs: Eduard Ayuso, Capucine Trollet
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
19:15
Welcome reception and Posters
Thursday 17 March
08:30-09:00
Registration
09:00-11:00
Session 3: Innovative approaches
Chairs: Michel Pucéat, Els Verhoeyen
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
11:30-13:30
Session 4: Cancer gene therapy
Chairs: Nicolas Boisgerault, Pierre Cordelier
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
15:00-16:45
Session 5 : Stem Cells and iPS
Chairs: John De Vos, Leila Maouche-Chrétien
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
17:15-19:15
Session 6: Towards clinical trials
Chairs: Anne Galy, Françoise Piguet
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
09:30-11:30
Session 7: Gene therapy & more
Chairs: Olivier Nègre, Juan Bueren
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
11:00-12:00
Closing keynote
Chairs: Els Verhoeyen
INV21 Juan Bueren, CIEMAT/Ciberer, Madrid ⓘ
Gene therapy in hematopoietic stem cell diseases: The Fanconi anemia model
Poster and oral presentation awards