3rd Spring School OXFORD UK

97 days

11 hours

17 mins

Changing the Face of Modern Medicine: Stem Cells & Gene Therapy

Detailed Scientific Programme: Sessions & Speakers

9:00 - 15:30  EDUCATION SESSION (program


14:00 - 15:35  MOLMED SYMPOSIUM (program)


16:00            OPENING SESSION

CHAIRS: Nathalie Cartier, Luigi Naldini, Nancy Witty

ROOM: Brunelleschi Auditorium

Welcome and Introduction: Nathalie Cartier, Luigi Naldini, Nancy Witty

INV23 Hans Clevers -  University Medical Centre Utrecht and Princess Maxima Center for pediatric oncology, Utrecht - Lgr5 stem cell-grown organoids and their applications

17:00              SESSION 1: Neural disease: modelling, reprogramming and transplantation in brain and retina

CHAIRS: Giuseppe Testa, Vania Broccoli

ROOM: Brunelleschi Auditorium

INV24 Fred Gage -  The Salk Institute, La Jolla, California - Modeling human psychiatric disease

INV25 Masayo Takahashi - RIKEN, CDB, Kobe -  Retinal cell using iPS cells

INV26 Malin Palmar -  Lund University - Towards a stem cell based therapy for Parkinson's disease

INV27 Paul Tesar -  Case Western Reserve University - Chemical approaches to oligodendrocyte remyelination



08:30              SESSION 2: Hematopoietic stem cells: from biology to clinical applications

CHAIRS: George Daley, Alessandro Aiuti

ROOM: Brunelleschi Auditorium

INV 28 John Dick -  Princess Margaret Cancer Centre, University Health Network, University of Toronto - How is human blood made?

INV 29 Leonard Zon - Boston Children's Hospital , Harvard Medical School - Using zebrafish to find new therapies for blood diseases

INV30 Luigi Naldini SR-Tiget, Milan - Advanced genetic engineering of hematopoiesis to treat human diseases

INV31 Marina Cavazzana -  Hôpital Necker- Paris Descartes - Gene therapy of inherited disease : advances and challenges

11:00              SESSION 2a: Imaging stem cells dynamics

CHAIRS: Fred Gage, Dominique Bonnet

ROOM: Boticelli

INV32 Valentina Greco -  Yale University - Tissue-scale coordination of cellular homeostatic and repair behaviors in live mice

INV33 Timm Schröder -  ETH Zürich - Long-term single cell quantification: new tools for old questions

OR01, Giulia CarolaInstitute of Biomedicine of the University of Barcelona (IBUB) - Altered functional activity in vmDA neurons derived from Parkinson’s disease-induced pluripotent stem cells (iPSC)  

OR02, Yein NamUniversity of Manchester - 3D-imaging and tissue reconstruction of deep-brain gene silencing with nanoscale, non-viral siRNA complexes

11:00              SESSION 2b: Eye stem cell and gene therapy

CHAIRS: Robin Ali, Alberto Auricchio

ROOM: Masaccio

INV34 Graziella Pellegrini Center for Regenerative Medicine-University of Modena - What does influence regeneration?

INV35 Deniz Dalkara - Inserm, UPMC Paris 6 AAV mediated gene therapy and beyond- maintaining and restoring vision

OR03  Anai Gonzalez Cordero - UCL - Generation and transplantation of human pluripotent stem cell derived cone photoreceptors into models of retinal degeneration

OR04 Jean-Philippe Combal - Gensight Paris - One-year follow-up study results after Intravitreal rAAV2/2-ND4 (GS010) injection in patients with vision loss due to G11778A ND4 Leber Hereditary Optic Neuropathy.

11:00              SESSION 2c: Central nervous system gene therapy

CHAIRS: Nathalie Cartier, Jerry Mendell

ROOM: Brunelleschi Auditorium

INV36 Alessandra Biffi - Gene Therapy Program Dana-Farber/Boston Children’s Cancer and Blood Disorders Center - HSC-based cell and gene therapy approaches for treating LSDs

INV37 Shin-ichi Muramatsu -  Division of Neurology, Department of Medicine Jichi Medical University - Gene therapy for neurodegenerative diseases

OR05, Claire Henchcliffe Weill Cornell Medical College  - Survival of embryonic tissue grafts in Parkinson’s disease: neuroimaging and clinical evidence at 17-18 years post-transplant

OR06, Nick Mazarakis – Imperial College London- PGC-1α overexpression by lentiviral vector attenuates amyloid-β load and neuronal loss in an Alzheimer’s disease model

12:45 - 13:45              Regulatory workshop for ATMP's

Martin Wisher: Regulatory guidance and advice on the Quality Control of Advanced Therapy Medicinal Products

14:00              SESSION 3: Skeletal and cardiac muscle stem cells: from biology and reprogramming to clinical applications

in memoriam Paolo Bianco

CHAIRS: Wim Fibbe, Fulvio Mavilio

ROOM: Brunelleschi Auditorium

INV38 Christine Mummery -  Leiden University Medical Center,  University of Twente - Challenges of immaturity and proliferation in using hPSC-derived cardiomyocytes as disease models

INV39 Mauro Giacca International Centre for Genetic Engineering and Biotechnology (ICGEB)  - Small RNA therapy for cardiac regeneration

INV40 Michael Laflamme University Health Network, Toronto - Remuscularization of injured hearts with human embryonic stem cell-derived cardiomyocytes

INV41 - Luis Garcia - Inserm UMR 1179 - Tricyclo-DNA: a new generation of antisense oligonucleotides for splice switching

16:30              SESSION 3a: Organoids and high throughput platforms

CHAIRS: Valentina Greco, Melissa Little

ROOM: Boticelli

INV42 Sergiu Pasca -  Stanford University - Generating 3D models of the human cerebral cortex to study development and disease

INV43 David  Tuveson - Cold Spring Harbor Laboratory - Detecting and killing pancreatic cancer

OR07 Francesco Saverio Tedesco - UCL London - Generation of Implantable 3D Skeletal Muscle Tissue from Human Embryonic Stem Cells and Muscular Dystrophy iPS Cells

OR08 Marianne Carlon, Laboratory for Molecular Virology and Drug Discovery, Division of Molecular Medicine, KU Leuven - The development and characterization of rAAV vectors in patient-derived intestinal organoids and CF mice as a treatment for cystic fibrosis

OR09 Emily Welby, UCL, Institute of Child Health, London -  Isolating and characterising human cone photoreceptors for cell therapy

OR10 Francesca Cavallo – University of Milan - Assay development and high throughput screening in iPSCs-derived cortical glutamatergic neurons from two neurodevelopmental disorders caused by symmetrical dosage imbalance.

16:30                   SESSION 3b: Stem cell based neural disease modelling

CHAIRS: Angela Gritti, Paul Tesar

ROOM: Masaccio

INV44 Giuseppe Testa, University of Milan  - Dissecting the genetic and environmental causes of chromatin dysfunction in autism and intellectual disability: an integrated platform of 2D and 3D stem cell-based models of neural development

INV45 Angel Raya, Center for Regenerative Medicine in Barcelona (CMRB) iPSC-based modeling of Parkinson’s disease

OR11, Pietro MazzaraSR-Tiget, Two factor fibroblast reprogramming generates induced Schwann cells with myelinogenic and nerve regenerating potential

OR12, Bianca Marchetti - University of Catania, Neural stem cell transplantation in Parkinsonian mice triggers an astrocyte-dependent dopaminergic neurorestoration

OR13, Matthew Ellit – Case Western Reserve University, Leveraging pluripotent stem cells as a scalable platform to discover chemical therapeutics for genetic disorders of myelin

OR14, Roxana Natt Medical University Innsbruck,  Mitochondrial disease phenotype in Friedreich’s ataxia patient iPSC-derived sensory neurons

16:30                   SESSION 3c: Cardiovascular gene and cell therapy

CHAIRS: Seppo Yla-Herttuala, Mauro Giacca

ROOM: Michelangelo

INV46 Seppo Yla-Herttuala, A.I.Virtanen Institute, Kuopio Therapeutic vascular growth for cardiovascular diseases

INV47 Andrew Baker, The University of Edinburgh - Non-coding RNA in vascular repair and regeneration

OR15, Ajan ReginaldCelixir Limited, Cardiff - Fixing the So-called Unfixable: Regenerating Untreatable Fixed Myocardial Scar in Heart Failure Patients

 OR16 Paula AlvesiBET/ITQB, Oeiras Using omics tools to improve differentiation and maturation of cardiomyocytes derived from human pluripotent stem cells

OR17 Cristina OlgasiUniversity of Piemonte, Correcting the bleeding phenotype in hemophilia A using lentivirally FVIII-corrected endothelial cells differentiated from hemophilic induced Pluripotent Stem Cells (iPSCs)

OR18 Virginie Lambert, INSERM U910 Aix Marseille Université , IMM| Institut Mutualiste Montsouris Paris , Progenitor cells seeded collagen patches migrate and differentiate through the failing RV myocardium : which benefit on the RV function ?

16:30                   SESSION 3d: Immunology/cancer immuno gene therapy I

CHAIRS: Chiara Bonini, Dirk Busch

ROOM: Brunelleschi Auditorium

INV48 David  Klatzman - Sorbonne Université, UPMC Univ Paris 06, INSERM umrs959; P-HP, Hôpital Pitié-Salpêtrière - The tolerant immune environment of tumors governs tumor fate and efficacy of immunotherapies

INV49 Waseem Qasim - University College London - First application of gene-edited ‘universal’ T cells for leukaemia

OR19, Margherita Norelli San Raffaele University Milan, Modeling the cytokine release syndrome and its treatment in a long-term xenotolerant mouse model of CAR-T cell immunotherapy

OR20, Paul Maciocia UCL Cancer Institute London, Targeting the TCR β-Constant Region for Specific Immunotherapy of T-Cell Malignancies

OR21, Samuel Rabkin, Massachusetts General Hospital, Harvard Medical School - Immunovirotherapy in combination with immune checkpoint inhibitors for treating glioblastoma stem cell-derived tumors

OR22, Maddalena NovielloSan Raffaele Scientific Institute , Multiple inhibitory receptors are expressed on central memory and memory stem T cells infiltrating the bone marrow of AML patients relapsing after allo-HSCT

18:30 - 20:30    Poster session I (Odd poster numbers)

Room: Leonardo and Fra Angelico

08:00              SESSION 4: Cancer immuno - gene therapy

CHAIRS: Katherine High, Attilia Bondanza

ROOM: Brunelleschi Auditorium

INV50 Gianpietro Dotti, Department of Microbiology and Immunology, UNC Chapel Hill CAR T-cell therapy: from CD19 to other targets

INV51 Stanley Riddell - Fred Hutchinson Cancer Research Center, University of Washington, Seattle -  Chimeric antigen receptor T-cells - killing cancer by design

INV52 Carl June - Center for Cellular Immunotherapies and Abramson Cancer Center, University of Pennsylvania, Children’s Hospital of Philadelphia, Novartis Institute for Biomedical Research, Cambridge, MA Engineering T-cells for cancer therapy

INV53 Chiara Bonini, Università Vita-Salute San Raffaele and Ospedale San Raffaele Scientific Institute TCR gene edited memory stem T cells for cancer immunotherapy

10:30              SESSION 4a: Haematopoietic stem cells and homeostasis

CHAIRS: Leonard Zon, Cynthia Dunbar

ROOM: Brunelleschi Auditorium

INV54 Emmanuelle Passegué,  University of California San FranciscoHSC, metabolism and fate decisions 

INV55 Michael Milsom, HI-STEM, DKFZ , Heidelberg  Hematopoietic stem cell attrition and regeneration in response to inflammatory stress.

OR23, Serena Scala, SR-Tiget Milan - Clonal tracking of hematopoietic stem and progenitor cells in vivo in humans

OR24, Els Verhoeyen, CIRI; Inserm U1111, Lyon - Measles virus glycoprotein pseudotyped lentivectors allow high-level transduction of pre-stimulated and resting HSCs and correct HSCs in total bone marrow from Fanconi Anemia patients

OR25, Olga Gan, University Health Network, Toronto, Ontario - Single-Cell Assay of Human Hematopoietic Stem and Progenitor Cells (HSPC) Following Gene Transfer Reveals Dramatic Heterogeneity in HSC Proliferative Potential and Points to a Role of Sphingolipids Metabolic Genes in HSC Function.

OR26 Makoto Otsu, Institute of Medical Science, University of Tokyo Multiple allogeneic progenitors in combination function as a unit to support early transient hematopoiesis following transplantation

10:30              SESSION 4b: MSC gene and cell therapy

CHAIRS: Michael Laflamme, Luis Garcia

ROOM: Boticelli

INV56 Wilfried Dalemans,  Tigenix NV, HasseltUse of expanded adipose stem cells in the treatment of inflammatory diseases.

INV57 Willem Fibbe,  Leiden University Medical Center    Therapeutic immune regulation by mesenchymal stromal cells

OR27, Maria Ester Bernardo, SR-Tiget Milan, Comprehensive characterization of bone marrow-derived mesenchymal stromal cells from patients affected by primary immunodeficiency

OR28, Sabine Geiger – Apceth GmbH & Co. KG, Munich, , Amelioration of lung function and pulmonary tissue regeneration after treatment with Alpha-1 antitrypsin (AAT)-expressing mesenchymal stem cells (MSCs) in a murine model of elastase-induced emphysema

OR29, Maria Fernandez Garcia- CIEMAT/CIBERER, IIS-FJD, UAM, Madrid, Mesenchymal stromal cells prevent graft failure in a mouse model of hematopoietic stem cell gene therapy

OR30, Hiudong Jia University of Bristol, Pulmonary artery reconstruction using cord blood-derived multipotent stem cells in vitro and in vivo study

10:30              SESSION 4c: In vivo Gene Therapy

CHAIRS: Xavier Anguela, Hildegard Buening

ROOM: Masaccio

INV58 Federicco Mingozzi Genethon Evry Assessing and modulating immunogenicity in AAV vector mediated gene transfer

INV59 Katherine High,  Spark Therapeutics Gene therapy for haemophilia

OR31, Terry Flotte, University of Massachusetts Medical School, Sustained expression with partial correction of neutrophil defects 5 years after intramuscular raav1 gene therapy for alpha-1 antitrypsin deficiency

OR32, Michela MilaniSR-Tiget, Vita-Salute San Raffaele University Milan, Liver-directed gene therapy with lentiviral vectors in animal models of hemophilia B

 OR33, Capucine Trollet Sorbonne Universités, UPMC Univ Paris 06, UM76, INSERM U974, Institut de Myologie, CNRS FRE3617, Towards Clinical Gene and Cell Therapies for OPMD

OR34, Giuseppa Piras, UCL Institute of Child Health, Red blood cells as therapeutic carrier in monogenic disorders

14:00              SESSION 5a: Cancer stem cells

CHAIRS: John Dick, Emmanuelle Passegue

ROOM: Masaccio

INV61 Dominique Bonnet,  The Francis Crick Institute, London Leukemic stem cell interactions with the microenvironment: friend or foe?

INV60 Michael Clarke, Stanford University - Stem cells in cancer and regenerative medicine

INV61 Inder Verma, , The Salk Institute, La Jolla, California , Plasticity of cancer cells: lessons from gliobastomas

OR35, Carolina Caserta: SR-Tiget,  Vita-Salute San Raffaele University, Milan, microRNA-126 Orchestrates a Stem Cell-like Program in Acute B Lymphoblastic Leukemia (B-ALL)

OR36, Robert Downey, Memorial Sloan Kettering Cancer Center - A stem cell oriented phylogeny of cancers derived novel cancer gene expression signature in all undifferentiated cancers as a therapeutic target

14:00              SESSION 5b: Ex vivo HSC based gene and cell therapy

CHAIRS: Juan Bueren, Guiliana Ferrari

ROOM: Brunelleschi Auditorium

INV63 Don Kohn -  University of California, Los Angeles - Gene Therapy for Primary Immune Deficiencies: ADA-SCID and XCGD.

INV64 Cynthia Dunbar - National Heart, Lung, and Blood Institute - Clonal expansion and long-term persistence of rhesus macaque NK cells with an adaptive phenotype as revealed by genetic barcoding

OR37, Harry Malech, National Institute of Allergy and Infectious Diseases - Lentiviral gene therapy with busulfan conditioning for older patients with SCID-X1

OR38, Susana Navarro Ordonez - CIEMAT/CIBERER, IIS-FJD, UAM, Madrid ,  Lentiviral-mediated Gene Therapy in Fanconi anemia A: Preclinical and First Clinical Studies

OR39, Ilana Moscatelli - : Lund University, Stem Cell Center - A clinically applicable lentiviral vector corrects NSG mice engrafting cells from patients with Infantile Malignant Osteopetrosis

OR40, Annamaria Aprile, SR-Tiget, Milan, University of Rome “Tor Vergata” , Thalassemic bone marrow microenvironment accelerates hematopoietic stem cell ageing and exhaustion

14:00              SESSION 5c: DNA based gene transfer and in vivo II

CHAIRS: Zoltan Ivics, Amber Salzman

ROOM: Boticelli

INV65 James Wilson,  University of PennsylvaniaAAV for liver directed genome editing

OR41, Nicola Brunetti Pierri, Tigem, Federico II University of Naples, Naples - Diversion Towards Non-Toxic Metabolites by Gene Transfer for Therapy of Primary Hyperoxaluria Type

OR42, Fanny Collaud Genethon, Evry, Development and production scale-up of an AAV8-UGT1A1 vector for the treatment of Crigler-Najjar syndrome

OR43, Oihana Murillo-SaucaCentro de Investigacion Medica Aplicada (CIMA) Pampona, Improvement of gene therapy for Wilson’s disease

OR44 Sabrina Triffault- Genosafe Evry- Monitoring of anti-drug antibody responses, from development to assay validation prior to clinical trial initiation. Focus on anti-AAV responses.

OR45, Irene Gil Farina, NCT DKFZ Heidelberg, Deciphering AAV vector persistence in hematopoietic progenitors

OR46, Mehdi Gasmi, Adverum Biotechnologies, Inc , Impact of Vector Design and Administration Technique in Gene Therapy for the Treatment of Age- related Macular Degeneration

16:30              SESSION 5: New technologies: targeted genome and epigenome editing, new vector design, organoids

CHAIRS: Christine Mummery, George Daley

ROOM: Brunelleschi Auditorium

INV66 Angelo Lombardo,  SR-Tiget, Vita-Salute San Raffaele University, Milan - Inheritable silencing of endogenous genes by hit-and-run targeted epigenetic editing

INV67 Feng Zhang, The Broad Institute, MIT, Cambridge, MassGenome engineering: prospects and challenges

INV68 Melissa Little, Murdoch Childrens Research Institute, University of Melbourne - Generating a kidney from human pluripotent stem cells: where to from here?

INV69 Pierre Vanderhaeghen, Université Libre de Bruxelles -  From pluripotent stem cells to cortical circuits

18:30 - 20:00    Poster Session II  (Even poster numbers)

Room: Leonardo and Fra Angelico

20:30 - 01:00    Molecular Mingle Party @ Mercato Centrale

09:00              SESSION 6a: RNA based gene transfer and integration studies  

CHAIRS: Eugenio Montini, Terry Flotte

ROOM: Michelangelo

INV71 Luca Biasco, SR Tiget, MilanGene Therapy Program Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, University College London - Clonal tracking of engineered human hematopoiesis through integration sites analysis

INV70 Christof von Kalle,  National Center for Tumor Diseases (NCT) Heidelberg,  - Wherever you go – there you are: tracking DNA modifications

OR47, Michael Rothe - Hannover Medical School  - New molecular surrogate assay for genotoxicity assessment (SAGA)

OR48, Monica VolpinSR-Tiget Milan, Identification and ranking of different chromatin insulators to block vector-driven enhancer-mediated insertional mutagenesis in vivo

09:00              SESSION 6b: Genome editing and gene correction

CHAIRS: Thomas Barnes, Jakub Tolar

ROOM: Brunelleschi Auditorium

INV72 Toni Cathomen,  University Medical Center Freiburg  - Highly efficient gene editing in hematopoietic stem cells

INV73 Charles Gersbach,  Duke University Genome editing for Duchenne muscular dystrophy

OR49, Caroline Kuo, University of California Los Angeles (UCLA)  - Targeted Gene Therapy in the Treatment of X-Linked Hyper-IgM Syndrome

OR50, Antonio Casini, University of Trento, Identification of high-fidelity Cas9 variants using a yeast-based screening

09:00              SESSION 6c: Cancer gene therapy

CHAIRS: Len Seymour, Inder Verma

ROOM: Masaccio

INV74 Ramon Alemany, Institut Catala d'Oncologia, IDIBELL-ICO Barcelona  - Cancer virotherapy with oncolytic adenoviruses

INV75 Yasutomo Nasu,  Okayama UniversityDevelopment of prostate cancer gene therapy in Japan

OR51, Doug Jolly Tocagen Inc, Clinical Update, Molecular Analyses, and Proposed Mechanism of Action of Toca 511 a Retroviral Replicating Vector in Three Ascending Dose Trials in Patients with Recurrent High-grade Glioma

OR52, Lukasz Kuryk - Targovax Oy, Helsinki, Anti-tumor potency of cancer vaccine ONCOS-102 in the treatment of malignant mesothelioma in pre-clinical and clinical studies

09:00              SESSION 6d: Immunology and allergy

CHAIRS: Federico Mingozzi, Ron Crystal

ROOM: Boticelli

INV76 Ron Crystal, Weill Cornell Medical College Gene therapy for hereditary and acquired life-threatening, immune-mediated disorders

INV77 Maria Grazia Roncarolo,  Stanford School of Medicine - Gene therapy-based approach for immune tolerance induction

OR53, Lucia Sereni, SR-Tiget, Vita-Salute San Raffaele University, Milan - Intrinsic defect in Was-/- platelets: studies in conditional mouse model and WAS gene therapy treated patients

OR54, Bruno Gaillet Université Laval, Quebec -  Gene therapy for Ebola virus infections based on AAV vectors and Zmapp antibody cocktail

10:30              SESSION 6: Gene therapy in the market

CHAIRS: Luigi Naldini, Sven Kili

ROOM: Brunelleschi Auditorium

INV78 Jonathan Appleby, GSK - Primary immune deficiencies: a natural target for ex vivo gene therapy

Alessandro Aiuti, SR Tiget Milan - Ex vivo gene therapy in ADA-SCID: clinical data and experiences to date

Sol Ruiz, AEMPS, Madrid - Translating experimental gene therapy into clinical reality

Julie Venners Christensen, GSK, Patient management through the gene therapy process

12:00              SESSION 7: In vivo gene therapy

CHAIRS: Olivier Danos, Amit Nathwani

ROOM: Brunelleschi Auditorium

INV82 Alberto Auricchio, Tigem, Naples -Gene therapy of mucopolysaccharidosis VI

INV83 Mark Kay, Stanford University Making a good vector even better: novel rAAVs for classical gene therapy and genome editing

14:30              SESSION 7a: Immunology/cancer immuno gene therapy II

CHAIRS: Pierre Cordelier, Ramon Alemany

ROOM: Brunelleschi Auditorium

INV84 Dirk Busch, Technische Universität München T-Cell and cancer immunotherapy

INV85 Bernard Gentner,  SR-Tiget , Milan Macrophage-based delivery of immunostimulatory and antiangiogenic molecules into the tumor microenvironment

OR55, Richard Morgan, bluebirdbio, Reduced CAR tonic signaling and methods to enhance memory T cells result in improved in vivo efficacy in human multiple myeloma xenograft models

OR56, Sarah Tettamanti, Universita' Milano Bicocca, Osp. San Gerardo/Fondazione MBBM, Monza , Balance of Anti-CD123 Chimeric Antigen Receptor (CAR) Binding Affinity and Density in an in vitro Model of Acute Myeloid Leukemia 

14:30              SESSION 7b: Gene silencing for small non-coding RNA's to epigenetic editing and gene disruption

CHAIRS: Tony Cathomen, Angelo Lombardo

ROOM: Boticelli

INV86 Michael Holmes,  Sangamo BioSciences  ZFN-mediated genome editing in the liver – towards the correction of lysosomal storage diseases

INV87 Frank Slack, Harvard Medical SchoolMicroRNA-based therapeutics in cancer

OR57, Salvatore Botta, Tigem Naples, Transcriptional silencing via synthetic DNA binding protein lacking canonical repressor domains as a potent tool to generate therapeutics

OR58, Belinda Cowling, IGBMC, Strasbourg, ASO-mediated Dnm2 knockdown prevents and reverts Myotubular myopathy in vivo in mice

14:30              SESSION 7c: Manufacturing of cell and gene therapy products

CHAIRS: Gabor Veres, Otto Merten

ROOM: Michelangelo

INV88 Paolo Rizzardi , MolMed S.p.A    - Challenges in vector and cell manufacturing in gene therapy

INV89 Ian Johnston, Miltenyi Biotec Mastering the challenges of manufacturing: the critical roles of closed systems and automation

OR59, Hildegard Büning, University of Cologne, DZIF, University Hospital Cologne, Hannover Medical School -  Improving the purity of Adeno-associated viral vector preparations using DNA minicircle technology

OR60, Melissa Bonner, bluebirdbio, Staurosporine Increases Lentiviral Transduction of Human CD34+ Cells

14:30              SESSION 7d: CNS gene therapy

CHAIRS: Alessandra Biffi, Nicole Deglon

ROOM: Masaccio

INV90 Nathalie Cartier,  INSERM U1169, MIRCEN CEA and Universite´ Paris-Sud, Universite´ Paris Saclay AAV-CYP46A1 brain administration restores cholesterol metabolism and is neuroprotective in Huntington’s disease

INV91 Jerry Mendell,  Nationwide Children’s Hospital, Ohio State University,  AVXS-101 phase 1 gene therapy clinical trial in SMA type 1

OR61 Brian Kaspar, Nationwide Children's Hospital, The Ohio State University Medical Center, AveXis, Inc - From bench to bedside: A novel approach in the treatment of SMA Type 1 with gene therapy

OR62 Taka Yamagata, Jichi Medical University, Improvements in motor function and PET findings following gene transfer to the patients with AADC deficiency

16:00             CLOSING

CHAIRS: Nathalie Cartier, Luigi Naldini

ROOM: Brunelleschi Auditorium

INV92 George Daley,  Boston Children's HospitalMilestones and barriers in hematopoietic stem cell derivation from pluripotent stem cells

ESGCT Outstanding achievement award

Amit Nathwani, Univeristy College London - Progress for gene therapy in haemophilia

ESGCT Young investigator award

OR63 Pietro Genovese, HS Tiget Milan Towards clinical translation of gene editing technologies for empowering adoptive immunotherapy or correcting inherited mutations

17:45              GERMLINE EDITING DEBATE

CHAIRS: Roberto Buccione

ROOM: Brunelleschi Auditorium

PARTICIPANTS TO THE DEBATE: Annelien Bredenoord, Giuseppe Testa, George Daley, Nathalie Cartier, Luigi Naldini

19:30: End of the debate and closing drinks