Wednesday, 03.04.2019


Coach pick up from hotel Renaissance & Oriente


Public engagement: Metta Pratt, WATS.ON, Oxford
Grant proposals and funding: Anne Galy, Genethon, Paris
Presentation skills: Graciana Diez-Roux, TIGEM Naples

12:00-13:00 Lunch (not provided by ESGCT)
13:15-15:15 Alberto Auricchio & Hildegard Büning
Welcome address
INV001 Andrea Ballabio, TIGEM, Naples
Opening keynote: The lysosome in health and disease
INV002 Juan Bueren, Ciemat/Ciberer, Madrid
Keynote: Gene therapy of inherited bone marrow failure syndromes
15:15-16:00 Coffee Break

Session 1: Spotlight on delivery tools
INV003 Axel Schambach, Hannover Medical School
The world of retroviruses and how it inspired approaches for gene therapy and genome surgery
INV004 Hildegard Büning, Hannover Medical School
Adeno-associated viral (AAV) vectors - How to train them for "the real life? 
INV005 Zoltan Ivics, Paul Elrich Institute, Langen
Turning genomic trash into treasure: transposons as tools for gene delivery
INV006 Anne Galy, Genethon, Paris
Immune responses to vectors administered in vivo with a focus on the use of lentiviruses

Historical talk on Pozzuoli area
18:30-22:00 Guided tour of Pozzuoli
Dinner in Pozzuoli
Coach back to Naples

Thursday, 04.04.2019

08:00 Coach pick up from hotel Renaissance & Oriente
Coach 1: Meet the expert in blood diseases: Giuliana Ferrari
Coach 2: Meet the expert in primary Immunodefficiencies: Alessandro Aiuti
Coach 3: Meet the expert in muscle gene therapy: Fulvio Mavilio
09:00-10:45   Session 2: Spotlight on stem cells/iPSC
INV007 Vania Broccoli, San Raffaele Scientific Institute, Milan
Generation of neurons from iPS/ES cells 
INV008 Giuliana Ferrari, SR Tiget, Milan
Haematopoietic stem cells - smart tools in gene therapy

Session 3: Gene and cell therapy of primary immunodeficiencies
INV009 Alessandro Aiuti, SR Tiget, Milan
From an idea to a product and beyond
10:45-11:15 Coffee break
11:15-13:00  Session 4: Gene and cell therapy for eye diseases
INV010 Alberto Auricchio, TIGEM, Naples
The retina as target for gene therapy
INV011 Robin Ali, University College London
Clinical trials of gene therapy for inherited blindness

INV012 Michele De Luca, University of Modena and Reggio Emilia
Keynote: Cell and gene therapy by somatic stem cells: the paradigm of epithelial stem cells
13:00-14:00 Lunch in canteen
14:00-15:00  Session 5: Spotlight on skeletal and cardiac gene therapy strategies
INV013 Mauro Giacca, ICGEB, Trieste
"How can you mend a broken heart” (Bee Gees, 1971):  meeting an old medical need with new genetic tools
INV014 Fulvio Mavilio, University of Modena and Reggio Emilia
Breathtaking therapies for patients who can’t breathe: the case of X-linked myotubular myopathy
15:00-15:30 Coffee break
15:30-17:30 Session 6: Spotlight on genome editing and epigenomic modifications
INV015 Pietro Genovese, SR Tiget, Milan
Genome editing in HSC
INV016 Angelo Lombardo, SR Tiget, Milan
Rewriting gene therapy through epigenetic editing
INV017 Anna Cereseto, University of Trento
Safer designer nucleases and their delivery

Round table
Challenges and hurdles of genome editing: ethics and regulatory affairs
Chair: Mauro Giacca, ICGEB, Trieste
Participants: Anna Cereseto, Angelo Lombardo, Pietro Genovese, Fulvio Mavilio
17:45-22:00 Coach back to Naples
Coach 1: Meet the expert in vector-mediated immune responses: Anne Galy
Coach 2: Meet the expert in AAV vectors: Hildegard Büning
Coach 3: Meet the expert in non-viral vectors: Zoltan Ivics

Sightseeing tour of Naples
Pizza in Naples

Friday, 05.04.2019

08:15 Coach pick up from hotel Renaissance & Oriente
Coach 1: Meet the expert in liver directed gene therapy: Gloria Gonzalez Aseguilonaza
Coach 2: Meet the expert in Cancer immunotherapy: Vincenzo Cerullo
Coach 3: Meet the expert in CNS gene therapy: Nathalie Cartier
09:30-11:00   Session 7: Gene therapy for the liver
INV018 Federico Mingozzi, Genethon, Paris
Gene therapy of haemophilia: past, present and future
INV019 Nicola Brunetti Pierri, TIGEM, Naples
Clinical trials of liver-directed gene therapy for inborn errors of metabolism
INV020 Gloria Gonzalez Aseguinolaza, FIMA, Pamplona
Gene editing for liver disease: primary hyperoxaluria
11:00-11:30 Coffee Break
11:30 - 12:30 Session 8: Gene and cell therapy for the CNS
INV021 Nathalie Cartier, ICM Paris
Gene therapy strategy for CNS
INV022 Alessandro Fraldi, TIGEM Naples
New strategies for CNS gene therapy
12:30-13:30 Lunch in canteen
13:30-15:00 Session 9: Immunotherapy and cancer
INV023 Vincenzo Cerullo, University of Helsinki
Dressing viruses in tumour’s clothing: welcome to the cloning-free oncolytic vaccine era
INV024 Chiara Bonini, SR Tiget, Milan
Cancer immunotherapy: beyond CAR-T cells
INV025 Antonella Folgori, Reithera, Rome
Innovative genetic vaccines for infectious diseases and cancer
15:00-15:30 Coffee break
15:30-17:30 INV026 Lucia Faccio, Telethon Foundation
Fondazione Telethon multiple stakeholder collaborative model to develop gene therapies

INV029 Guangping Gao, Umass Medical School, Worcester, MA
Keynote: Human gene therapy: the road to today and beyond

INV028 Luigi NaldiniSR Tiget, Milan
Closing keynote: Nearly 3 decades of gene and cell therapy - has gene therapy fulfilled its promise?
Closing: Hildegard Büning and Alberto Auricchio

Coach back to Naples