Print Programme
Tuesday 22 October 2019
17:00-19:00

 
2019 Opening session
Chairs: Hildegard Büning, Fàtima Bosch, Angel Raya

Room 113-117

INV025: Andrea Ballabio
Tigem, Naples
The lysosome: main regulator of cell metabolism and attractive therapeutic target

INV026: Masayo Takahashi
Riken, Kobe
The benefits and risks of iPSC-derived retinal cell therapy
19:00-20:00 Welcome reception
 
Wednesday 23 October 2019
08:30-10:30
Session 1a: Gene and cell targeting

Chairs: Toni Cathomen, Thierry VandenDriessche
Room 116-117

INV027: Els Verhoeyen
EVIR/CIRI/INSERM U1111, Lyon
Site specific genome editing in human T, B cells and HSCs using baboon envelope gp pseudotyped “Nanoblades” loaded with Cas9/sgRNA and specific knock-in in HSCs when combined with an AAV-6 encoding donor DNA. 

INV028: Pietro Genovese
SR Tiget, Milan
“One size fits all" strategy for T cell correction, selection and depletion as new Treatment for HIGM1 syndrome

OR01: Laura Torella
CIMA, Pamplona
Paired-nickase S.aureus Cas9 system is an efficient and potentially safer in vivo treatment for Primary Hyperoxaluria type 1

OR02: Antonio Carusillo
University of Freiburg
HDR-CRISPR: a novel system to promote Cas9-mediated homology-directed DNA repair

OR03: Alessia Cavazza
University College London
Targeted gene correction of human haematopoietic stem cells for the treatment of Wiskott-Aldrich Syndrome

OR04: Tiziana La Bella 
Inserm U1138, Paris
Adeno-associated virus in human liver: natural history and consequences in tumor development
Session 1b: Innovaties therapies in cancer
Chairs: Vincenzo Cerullo, Ramon Alemany
Room 113-115

INV029: Cristina Fillat
Idibaps, Barcelona
Pancreatic cancer: from target discovery to innovative therapies

INV030: Chiara Bonini
Ospedale San Raffaele, Milan
Genome editing of memory stem T cells for cancer immunotherapy

OR05: Sheena Smith
University of Zurich
Paracrine delivery of therapeutic biologics for cancer

OR06: Olivier Humbert
Fred Hutchinson Cancer Research Centerm Seattle, WA
Base editor-mediated CD33 engineering to improve safety and efficacy of CD33-targeted cancer therapy


 
Session 1c: iPSC & organoids

Chairs: Josep M Canals, Axel Schambach
Room 112



INV032: Nico Lachmann
Hannover Medical School
The "new stem cell“: macrophages for disease modeling
and cell based therapies

INV033: Nuria Montserrat
IBEC Barcelona
Engineering human pluripotent stem cells for organoid applications in regenerative medicine

INV034: Alfonso Martinez-Arias
University of Cambridge
Gastruloids: an ESC based model for mammalian gastrulation and axial
organization

OR07: Frank Staal
Leiden University Medical Center
De novo generation of functional human thymus organoids from induced pluripotent stem cells

OR08: Patricia Peking
Paracelsus Medical University, SCI-TReCS, Salzburg
Self-assembly of human stem/progenitor cells creates neo-vascularized skin and skin organoids
Session 1d: ATMP manufacturing

Chairs: Philippe Moullier, Francesc Godia
Room 211


INV035: Richard Snyder
Thermo Fisher Scientific, Cambridge, MA
Viral vector manufacturing and testing

INV036: Matthias Hebben
LogicBio, Cambridge, MA
AAV manufacturing: critical parameters influencing vector quality attributes.
 
INV037: Hanna Lesch
Kuopio Center for Gene and Cell Therapy,
Translational research of AAV expressing VEGF-B: large scale manufacturing  process development, analytical development and pre-clinical studies in ischemic porcine model

INV038: Amine Kamen
McGill University, Montreal
Process intensification for manufacturing of viral vectors for cell and gene therapy

INV039: Luca Alberici
Molmed, Milan
20 years journey of viral vector manufacturing and cell engeneering

INV040: Carol Knevelman 
Oxford BioMedica
Efficient processes for the commercial manufacture of lentiviral vectors

OR09: Ana Filipa Rodrigues
iBET, Oeiras
Cell- and vector-engineering approaches for manufacturing high-titer GaLV pseudotyped lentiviral vectors from stable and constitutive producer cell lines

OR10: Vinicia Polito
Bambino Gesù Children’s Hospital, Rome
Generation of an automated GMP-grade protocol in a closed system for the expansion of polyclonal memory γδ-T cells for a “third party” cell bank. 
10:30-11:00 Coffee break
11:00-13:00
 
          Plenary session 2: From Pre-clinical to clinical gene therapy
Chairs: Juan Bueren, Alessandro Aiuti
Room 113-117



INV041: Steve Brown
IMPC, Harwell
The International Mouse Phenotyping Consortium (IMPC): Delivering a comprehensive catalogue of mammalian gene function and novel pre-clinical models

INV042: Fàtima Bosch
UAB, Barcelona
Translational gene therapy approaches to treat Mucopolysaccharidosis

INV043: Matthew Porteus
Stanford University, CA
Engineering of cells using genome editing to treat disease

INV082: Angelo Lombardo
SR Tiget, Milan
Exploiting targeted epigenome editing for therapeutic and biological applications
13:00-15:00 Lunch & Poster session I
13:45
14:45
Meet the society leadership
Registration area

A chance to meet the leadership of all national and international societies
15:00-17:00
 
Plenary session 3: Gene therapy clinical trials I
Chairs: Adrian Thrasher, Seppo Yla Herttuala
Room 113-117

INV045: David Williams
Harvard Medical School, MA
Validation of BCL11A as molecular target to treat sickle cell disease: results of pilot and feasibility trial using shmiR technology

INV046: Fulvio Mavilio
Audentes Therapeutics, San Francisco, CA
Gene therapy for X-Linked Myotubular Myopathy

INV047: Alberto Auricchio
TIGEM, Naples
Gene therapy of mucopolysaccharidosis type VI

INV048: Jude Samulski
UNC Gene Therapy Centre, Chapel Hill
The road from Canavan to Pompe - a 20 yr clinical journey
17:00-17:30 Coffee break
17:30-19:30 Session 2a: Skeletal muscle & bone gene therapy
Chairs: Marinee Chuah, Ana Buj Bello
Room 116-117



INV049: Thierry VandenDriessche
VUB, Brussels
Gene therapy flexes its muscles

INV050: Pura Muñoz
Universidad Pompeu Fabra, Barcelona
Muscle stem cell aging and rejuvenating strategies

INV051: Johan Richter
Lund University
Towards haematopoietic stem cell-targeted gene therapy of infantile malignant osteopetrosis

OR11: Daniel Moore
University College London

Modelling skeletal muscle laminopathies with human ips cells and bio-engineered skeletal muscles: prospects for genetic therapies


OR12: Magdalena Matloka
Center of Research in Myology, Paris

Decoy-based gene therapy for Myotonic Dystrophy

Session 2b: First in man gene therapy
Chairs: Alessandra Biffi, Giuliana Ferrari
Room 113-115


INV052: Claire Booth
UCL, London
Evolving haematopoietic stem cell gene therapy

INV053: Alessandro Aiuti
SR Tiget, Milan
Haematopoietic stem cell gene therapy for MPSIH

INV054: Terrence Flotte
University of Massachusetts Medical School, MA
First-in-human gene therapy for Tay-Sachs disease: Report of two infants treated on an expanded access clinical trial of rAAVrh8-HexA/HexB (AXO-AAV-GM2)

OR13: Erika Zonari
SR TIGET, Milan

Shortened ex vivo transduction for haematopoietic stem cell gene therapy of Hurler disease: impact on haematopoietic reconstitution potential 

OR14: Gavin Corcoran
Axovant Sciences, NYC

The SUNRISE-PD Study, a clinical trial of AXO-LENTI-PD: a CNS-directed gene therapy for the treatment of Parkinson’s Disease

Session 2c: Next generation engineered T&NK cells
Chairs: Sonia Guedan, Boris Fehse
Room 112

INV055: John Maher
UCL, London
T4 CAR T-cell immunotherapy of head and neck cancer – Phase I trial update

INV056: Pablo Menendez
Josep Carrera Research Center, Barcelona 
Advancing adoptive cellular immunotherapy for acute leukemia

IN0V57: Concetta Quintarelli
Ospedale Bambino Gesù, Roma
Harnessing NK lymphocytes: an alternative platform for allogeneic CAR therapy

OR15: Frederic Thalheimer
Paul-Ehrlich Institut, Langen

In vivo generated human CAR T cells eradicate B cell leukemia in preclinical mouse models

OR16: Arpad Szoor
University of Debrecen
Trastuzumab derived HER2-specific CAR for trastuzumab-resistant breast cancer: CAR T-cells successfully engage target epitopes that are not accessible to antibodies

Session 2d: AAV vector development
Chairs: Els Verhoeyen, Jim Wilson 
Room 211

INV058: Hildegard Büning
Hannover Medical School
Advanced AAV vectors - capsid engineering and beyond

INV059: Eric Kelsic
Harvard Medical School, MA
Machine-guided AAV capsid engineering for gene therapy

INV060: David Schaffer
University of California, Berkeley, CA
Directed evolution of novel AAV vectors for clinical gene therapy

OR17: Jihad El Andari
Heidelberg University Hospital

Semi-rational engineering of next-generation AAV capsids for muscle gene therapy

OR18: Eric Pozsgai
Sarepta Therapeutics, Cambridge, MA
Systemic gene transfer with AAVrh74.MHCK7.SGCB increased β-sarcoglycan expression in patients with limb girdle muscular dystrophy type 2E

 
Thursday 24 October 2019
8:30-10:30

 
Session 4: Gene therapy clinical trials II
Chairs: Virginia Haurigot, Fulvio Mavilio
Room 113-117

INV061: Juan Bueren 
CIEMAT CIBERER, Madrid
Gene therapy for patients with Fanconi anaemia

INV062: Michel Sadelain
Memorial Sloan Kettering Cancer Center, NY
Clinical strategies to overcome resistance to CAR therapy

INV063: Don Kohn
UCLA,  CA
Haematopoietic stem cell gene therapy for sickle cell disease    

INV064: Steven Gray
UT Southwestern, Dallas, TX
Intrathecal AAV9 as a platform approach to treat multiple CNS disorders
10:30-11:00 Coffee break
11:00-13:15

 
Presidential symposium
Chairs: Hildegard Büning, Angel Raya
Room 113-117



INV065: Hildegard Büning
Hannover Medical School
Presidential address

INV066: Hans Peter Kiem
Fred Hutchinson Cancer Research Center, Seattle, WA
Targeting distinct haematopoietic stem cell populations for ex vivo and in vivo gene therapy


ESGCT Outstanding achievement award 

INV067: Nathalie Cartier Lacave
Gene therapy strategies for neurodegenerative diseases : From genetic to complex diseases


ESGCT Young Investigator award

0R51: Paula Rio
CIEMAT CIBERER, Madrid
Innovative haematopoietic gene therapy strategies in Fanconi anaemia


ESGCT AGM
13:15-14:45 Lunch & Poster session II
13:45
14:45
Ethics in gene and cell therapy
Room 112

Chair: Odile Cohen-Haguenauer, Bernd 
Gänsbacher
Odile Cohen-Haguenauer, Hôpital Saint Louis, Paris
Ethics of first-in-man clinical trials in gene therapy

Nicholas Anagnou, University of Athens
Ethical issues of unproven therapies and stem cell tourism

Matthew Porteus, Stanford University, CA
The strict criteria for germline editing proposed by the 2017 study committee

François Moreau-Gaudry, University of Bordeaux
CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations

Roundtable discussion
Infectious diseases meet gene therapy
Room 211

Chair: Hans Pieter Kiem, Monique Nijhuis
 
Javier Martinez-Picado, IrsiCaixa, Barcelona
The IciStem experience: learning from allogeneic stem cell transplantation to cure HIV

Anne Galy, Genethon, Evry
Vectored immunoprophylaxis of infectious diseases : Current efforts and future perspectives

Toni Cathomen, University of Freiburg
The prospect of genome and epigenome editing to cure HIV and other infectious diseases

Roundtable discussion
14:45-16:45 Session 3a: Latest news from cardiovascular and pulmonary gene therapy
Chairs: Terry Flotte,
Mauro Giacca

Room 116-117

INV068: Mauro Giacca
Kings College London 
Identification of novel cardioprotectors from AAV libraries encoding for the secretome

INV069: Maria Blasco
CNIO, Madrid 
Telomerase gene therapy as a novel treatment for pulmonary fibrosis

INV070: Seppo Ylä Herttuala
University of Eastern Finland, Kuopio 
Recent developments in cardiovascular gene therapy

OR19: Rosario Perona
CSIC/UAM, CIBER, Madrid
GSE4, a Dyskerin derived peptide enhances pneumocyte growth, reduces apoptosis, DNA damage and lung fibrosis.

OR20: Rafael Sànchez-Sànchez
Instituto de Investigacion sanitaria La Fe, Valencia
Cardioprotective effect of miRNAs derived from mesenchymal stem cells extracellular vesicles in doxorubicin-induced damage

 
Session 3b: Sensory and CNS gene therapy I
Chairs: Alberto Auricchio, Françoise Piguet
Room 113-115

INV071: Alessandra Biffi
Harvard Medical School, MA
HSC gene therapy for neurometabolic and neurodegenerative disorders

INV072: Luk Vandenberghe
Harvard Medical School, MA
AAV discovery for neurosensory indications and beyond.

INV073: Hinrich Staecker
The University of Kansas, Lawrence, KS
Strategies for translating inner ear gene therapy; lessons from the first in human clinical trial

OR21: Szilárd Kiss
Weill Cornell Medical College, NY
Intravitreal gene therapy with ADVM-022 for neovascular age-related macular degeneration (phase 1 OPTIC Trial)

OR22: Adrian Thrasher
University College London
Phase 2/3 trial to assess the safety and efficacy of Lenti-D autologous haematopoietic stem cell gene therapy for cerebral adrenoleukodystrophy
Session 3c: Immune responses to gene therapy
Chairs: Federico Mingozzi, Anne Galy
Room 112

INV074: Percy Knolle
TUM, Munich
Mechanisms of liver tolerance and implication for liver-targeted gene therapy

INV075: Roland Herzog
Indiana University, Bloomington, IN
Immune response mechanisms against AAV vectors

INV076: Anna Kajaste-Rudnitski
SR Tiget, Milan
Vector-host interactions and innate immunity in haematopoietic stem cell gene engineering

OR23: Giorgia Squeri
SR Tiget, Milan
Targeting pre-existing anti-transgene T cell response for effective gene therapy of Mucopolysaccharidosis type-I 

OR24: Malo Journou
University of Nantes INSERM 1089
Recombinant AAV-mediated gene transfer to the skeletal muscle is associated to immune modulation of transgene expression in the macaque model

 
Session 3d: Blood diseases
Chairs: Guillermo Guenechea, Francisco Martin
Room 211

INV077: Jose Carlos Segovia Sanz
CIEMAT CIBERER Madrid
Treatment of Pyruvate Kinase Deficiency: From the laboratory to the lentiviral gene therapy clinical trial

INV078: Giuliana Ferrari
SR TIGET Milan
Gene therapy for transfusion dependent beta-thalassemia: A lesson from patients

OR25: Yari Gimenez Martinez
CIEMAT/CIBERER, Madrid
Lentiviral-mediated Phenotypic correction of CD34+ cells from RPS-19-deficient Diamond-Blackfan aneamia patients

OR26: Denise Klatt
Hannover Medical School
Competitive sgRNA screen identifies Mapk14 as a druggable target to improve HSPC engraftment in a proinflammatory environment

OR27: Evangelia Yannaki
G. Papanikolaou Hospital, Thessaloniki
Interim results from the phase 3 Northstar-2 and Northstar-3 studies of LentiGlobin gene therapy for the treatment of transfusion-dependent β-thalassaemia

OR28: Serena Scala 
SR Tiget, Milan
Haematopoietic reconstitution dynamics of MPB- and BM-derived haematopoietic stem/progenitor cells in Wiskott-Aldrich syndrome patients treated with lentiviral gene therapy
16:45-17:15 Coffee break
17:15-19:15

Room
113-117
Plenary session 5: Gene editing, toxicity
Chairs: Robin Ali, Luigi Naldini
Room 113-117



INV079:David Liu 
Harvard University, MA
Prime Editing: Search-and-replace genome editing without double-strand breaks or donor DNA

INV080: David Rawlings
University of Washington, Seattle, WA
Gene editing in human T and B cells for novel clinical applications

INV081: Jim Wilson
University of Pennsylvania, Philadelphia, PA
Safety and efficacy of in vivo editing in primate liver

INV044: Luigi Naldini
SR Tiget, Milan 
Advanced genetic engineering of haematopoiesis to treat human diseases
20:00 Molecular Mingle
MNAC, Barcelona
Coaches from CCIB at 19.15

          
 
Friday 25 October 2019
09:00
11:00

 
Session 4a: Sensory and CNS gene therapy II
Chairs: Nathalie Cartier, Jose Toledo Aral
Room 116-117


INV083: Abdel Benraiss
University of Rochester, NY
Cell replacement strategies for striatal regeneration in Huntington’s disease

INV084: Robin Ali
UCL, London
ES-derived cone photoreceptor transplantation for the treatment of retinal degeneration 

OR29: Bence Gyorgy
Institute of Molecular and Clinical Ophthalmology Basel
Allele-specific gene disruption through discrimination of a single base change by S. aureus Cas9-KKH prevents progressive hearing loss after AAV-mediated gene delivery

OR30: Allison Bradbury
University of Pennsylvania, Philadelphia, PA
Intrathecal AAV9-GALC corrects both central and peripheral nervous system disease in canine Krabbe disease in a dose and time-dependent manner

OR31: Pengcheng Zhou
Voyager Therapeutics, Cambridge, MA
Widespread, dose-dependent and durable huntingtin lowering in Huntington’s disease-relevant brain regions of large mammals with intraparenchymal administration of the AAV gene therapy VY-HTT01

OR32: Kevin Foust
AveXis, San Diego, CA
Intrathecal administration of AVXS-301 for amyotrophic lateral sclerosis (ALS): survival extension and SOD1 reduction in mice and nonhuman primates
Session 4b: New approaches in gene editing
Chairs: Paula Rio, Jose Carlos Segovia
Room 113-115


INV085: Juan Rodriguez-Madoz
University de Navarra, Pamplona
Disease modelling and genome editing for treatment of rare metabolic diseases.

INV086: Claudio Mussolino
University of Freiburg
Advances in precision genome and epigenome editing for human gene therapy

OR33: Edoardo Schneider
IGCEB, Trieste
Human microRNAs selected by high-throughput screenings enhance CRISPR/Cas9-based homologous recombination in post-mitotic hearts

OR34 Laura Ugalde Díaz
CIEMAT/CIBERER, Madrid
Broad applicability of NHEJ-mediated gene editing to correct mutations in a variety of Fanconi anaemia genes.
Session 4c:
Metabolic and genetic diseases
Chairs: Steven Gray, Fatima Bosch
Room 112

INV087: Federico Mingozzi,
Spark Therapeutics, Philadelphia, PA
Liver gene transfer as a new modality for enzyme replacement therapy, the example of Pompe disease

OR35: Petr Ilyinskii
Selecta Biosciences, Watertown, MA
ImmTOR™ tolerogenic nanoparticles enhance transgene expression after both initial and repeat dosing in a mouse model of methylmalonic acidemia treated with Anc80 AAV-Mut vector

OR36: Heather Gray-Edwards
University of Massachusetts, Boston, MA
Bicistronic AAV gene therapy for Tay-Sachs disease

OR39: Maria Jose de Castro
Hospital Universitario Santiago de Compostela
Safety, tolerability, biopotency and neurocognitive data of ABO-102 in Transpher A, an open-label, multicenter, single-dose, dose-escalation, Phase 1/2 Clinical Trial in Sanfilippo Syndrome type A (Mucopolysaccharidosis IIIA

OR40: Verónica Jiménez
UAB, Barcelona
AAV-FGF21 gene therapy mediates healthspan expansion in old mice 



 
Session 4d: Regulatory session
Chairs: Martina Schüssler-Lenz, Ilona Reischl
Room 211





INV088: Martina Schüssler-Lenz
Paul-Ehrlich-Institut, Langen
The EU landscape of authorised advanced therapies: presence and future

INV089: Heli Suila
Finish Medicines Agency
Quality considerations for gene therapies

INV090: Anne Engedahl Pastoft
Danish Medicines Agency
Non-clinical 84
data supporting gene therapy clinical trials’

INV091: Jan Müller-Berghaus
Paul-Ehrlich-Institut, Langen
Scientific and regulatory support for gene therapy in european countries

Q&A and panel discussion with chairs and speakers, Patrick Celis (European Medicines Agency), audience
11:00-11:30 Coffee break
11:30-13:30
Session 5a: Cancer gene therapy
Chairs: Cristina Fillat, Javier Garcia-Castro 
Room 116-117


INV092: Juan Fueyo
MD Anderson, Houston, TX
Virus, immunology and gliomas: new paradigms

INV093: Ramon Alemany
ICO, Barcelona
Potential and limitations of oncolytic adenoviruses

INV094: Vincenzo Cerullo
University of Helsinki
Dressing viruses in tumor’s clothing: cloning-free platforms to trigger tumor-specific immune response

OR41: Daniel Abate - Daga
Moffit Institute, Tampa, FL
TBC

OR42: Maria Cristina Ballesteros
CIMA, Pamplona
Local expression of a PD-L1 blocking antibody from a self-replicating RNA vector induces potent antitumor responses
Session 5b: New delivery systems and technologies
Chairs: Ander Izeta, Zoltan Ivics
Room 113-115

INV095: Helmut Hanenberg
University of Duisburg - Essen
Genetic gold for the treatment of haemophilia

OR43: Jason Wright
Homology Medicines Inc. Bedford, MA
Nuclease-free genome editing by AAVHSC vectors leads to in vivo genome correction and amelioration of disease phenotype in a mouse model of phenylketonuria (PKU)

OR44: Jonathan Finn
Codiak BioSciences, Cambridge, MA
A versatile platform for precision exosome engineering

OR45: Manel Llado 
TIGEM, Naples
Homology-independent targeted integration to counteract toxic gain-of-functions and loss of transgene expression due to cell proliferation.

OR46: Stefanie Gehrig-Giannini
Bayer AG Preclinical Research, Köln
Engineering potent, small, chimeric, synthetic, RNA-guided nucleases (sRGN) from four uncharacterized Cas9 genes

OR47: Giandomenico Turchiano
University College London
CAST-Seq, a novel preclinical genotoxicity assay, enables qualitative and quantitative insights in chromosomal aberrations in gene-edited human haematopoietic stem cells

OR48: Anthony Forget
Intellia Therapeutics, Cambridge, MA
In vivo gene knockout followed by targeted gene insertion results in simultaneous reduced mutant protein levels and durable transgene expression
 
Session 5c: Liver gene and cell therapy
Chairs: Gloria Gonzalez-Aseguinolaza, Xavier Anguela
Room 112

INV096: Paolo Martini
Moderna Therapeutics, Cambridge, MA
Messenger RNA therapy for inherited metabolic disorders

INV097: Nick Weber
Vivet Therapeutics, Pamplona
Targeting the liver to fix the bile: Opportunities and challenges in gene therapy for inherited cholestasis

OR37: Leah Venturoni
National Human Genome Research Institute, Bethesda, MD
Treatment of methylmalonic acidemia (MMA) by targeted integration of MMUT into Albumin with a promoterless AAV vector (GeneRideTM) confers a progressive hepatocellular growth advantage in mice

OR38 David Rodriguez- Buritica
The University of Texas, Dallas, TX
Positive cohort 1 results from the phase 1/2, AAV8-mediated liver-directed gene therapy trial in glycogen storage disease type Ia (GSDIa)

OR49: Ana Cristina Puga
Ultragenyx, Novato, CA
A phase 1/2 clinical trial of AAV8 gene therapy in adults with late-onset OTC deficiency: CAPtivate cohort 1 + 2 results

OR50: Anais Amaya
Children's Medical Research Institute, Westmead Inhibition of proliferation in primary human hepatocytes following in vivo AAV-mediated genome editing
Session 5d:
Make gene therapy a clinical reality
Chairs: Alessandro Aiuti, Hildegard Büning
Room 211

INV098: Alessandro Aiuti
SR TIget, Milan
Turning blood stem cells into medicinal products: lessons learned and future challenges

INV099: Serge Braun
AFM, Paris
Patients are not just patients, they are also pioneers. The AFM-Telethon perspective

INV100: Sol Ruiz
Spanish Medicine Agency, Madrid
Regulation of ATMP in the EU: Take it easy

INV101: Magda Papadaki
Merck, London
Reinventing the system for ATMPs: need for integrated development, assessment and business models
13:30-15:30 13:30 - 14:00 - SETGYC General Assembly (Room 113-115)  
Lunch
14:00
15:00
Current challenges of the AAV vectors
Room 211

Chairs: Hildegard Büning, Dirk Grimm


Tiziana La Bella, Genethon, Evry 
Genotoxicity - Is there a danger?

Hildegard Büning, Hannover Medical School Pre-existing and de novo immune responses towards AAV - what do we know and how can we manage this challenge?

Dirk Grimm, University of Heidelberg 
How to deal with AAVs lack in cell type specificity and efficiency - capsid engineering as possible solution

Eduard Ayuso, University of Nantes
How to produce clinical grade AAV vectors large scale

Panel Discussion: Hildegard Buning, Dirk Grimm, Eduard Ayuso, Tiziana La Bella, Terry Flotte
Current challenges of Lentiviral vectors 
Room 112

Chairs: Els Verhoeyen, Axel Schambach


Jose Carlos Segovia, CIEMAT/CIBERER, Madrid
Expanding the range of gene therapy to challenging diseases

Els Verhoeyen, EVIR/CIRI/INSERM U1111, Lyon
The future: In vivo gene therapy targeting haematopoietic stem cells and T cells

Pietro Genovese, Harvard Medical School
Gene editing: Towards clinical applications

Hans Pieter Kiem, Fred Hutchinson Cancer Research Center, Seattle, WA
Challenges in translating gene therapy to more patients and countries

Panel Discussion: Anne Galy, Paula Rio, Els Verhoeyen, Axel Schambach, Jose Carlos Segovia, Pietro Genovese, Hans Pieter Kiem
15:30-17:30 Plenary session 6: Challenges for the future
Chairs: Fatima Bosch, Gerry McLachlan
Room 113-117



INV102:Olivier Rabin
WADA, Montreal
Gene doping: The ultimate deviation of gene therapy

INV103: Carl June
University of Pennsylvania, Philadelphia, PA
Updates in CAR T cell therapies

Keynote INV104: Kathy High
Spark Therapeutics, Philadelphia, PA
Turning genes into medicines: Challenges for the next 5 years and beyond

Closing remarks
17:30-18:00 Closing drinks