Friday 14 October
08:30-09:00
Registration
09:00-10:30
Pentland /Sidlaw
Parallel 6a
Gene & epigenetic editing III
Chairs: Toni Cathomen, University of Freiburg / Axel Schambach, Hannover Medical School
INV54: Luigi Naldini, SR-Tiget, Milan
Transformative Approaches to Genetic Engineering of Hematopoiesis based on Gene Editing and RNA Transfection
OR61: Ana Hinckley Boned, Genyo, Granada
A negative regulatory region in WAS intron 1 controls megakaryocytic differentiation
OR62: Enrico Surace, University of Naples
Therapeutic changes of cis and trans regulatory elements
OR63: Lei Lei, University of Freiburg
Base editing in hematopoietic cells corrects the disease underlying intronic mutation in a mouse model of familial hemophagocytic lymphohistiocytosis
OR64: Samuele Ferrari, SR-Tiget
Assessing Stealth and Sensed Base and Prime Editing in Human Hematopoietic Stem/Progenitor Cells
09:00-10:30
Fintry
Parallel 6b
Novel advances in gene and cell therapy in the US
Chairs: Rayne Rouce, Texas Children's Hospital
INV49: Stephen Russel, Mayo Clinic, Rochester
Clinical experience with VSV-IFNb-NIS, an oncolytic vesicular stomatitis virus.
INV50: Rayne Rouce, Texas Children's Hospital
Next Level Cell Therapy": New Targets, New Sources, New Possibilities...New Problems
INV51: Rebecca Larson, Massachusetts General Hospital and Harvard Medical School.
CAR T cells in solid and liquid tumors: differences in cytotoxicity and cell adhesion.
09:00-10:30
Lomond Suite
Parallel 6c
CNS and sensory III
Chairs: Giulia Massaro, UCL London / Nicole Deglon, University of Lausanne
INV52: Stephan Pollard, University of Edinburgh
Synthetic super-enhancers enable cell type-selective expression of anticancer payloads for viral gene therapy
INV53: Stefanie Schorge, University College London
OR59: Pasqualina Colella, Stanford University
Novel conditioning enables high and stable repopulation of macrophage/microglia niches by wild type and genome-edited hematopoietic cells.
OR60: Annita Montepeloso, Harvard Medical School
Targeting CX3CR1 Gene To Improve Microglia Reconstitution And Transgene Delivery Into The CNS Upon Hematopoietic Stem And Progenitor Cell Transplant
10:30-11:00
Break
11:00-13:00
Pentand / Sidlaw
Parallel 7a
Cardiovascular diseases
Chairs: Stuart Nicklin, University of Glasgow / Seppo Ylä-Herttuala, University of Eastern Finland, Kuopio
INV63: Wolfram Zimmermann, University Medical Center Göttingen
Tissue Engineered Heart Repair from Bench to Bedside
INV64: Andrew Baker, University of Edinburgh
Modification of vein graft failure by gene therapy and RNA therapeutics
INV65: Roger Hajjar, Ring Therapeutics, Cambridge MA
Clinical Cardiac Gene Therapy Comes of Age
OR67: Ignacio Perez de Castro, Instituto de Salud Carlos III
Study of the potential of gene therapy approaches for the treatment of LMNA-related congenital muscular dystrophy
OR68: Izabella Kraszewska, Jagiellonian University
Identification of barriers to AAV transduction in the heart: focus on receptor availability and intracellular vector processing
11:00-13:00
Fintry
Parallel 7b
Nanoparticles and
biomaterials
Chairs: Alexander Baker, Accession Therapeutics, Oxford / Els Verhoeyen, Université de Nice, Université de Lyon
INV60: Zoltan Ivics, Paul Ehrlich Institute
Advances in Sleeping Beauty transposon-based therapeutic cell engineering
INV61: Ivana Trapani, Tigem, Naples
Tackling diseases due to mutations in large genes using AAVs
INV62: Helder Santos, University of Helsinki
OR65: Keittisak Suwan, Imperial College London
Chimeric phagemid/AAV for targeted gene delivery and cancer immunotherapy
OR66: Michele Palamenghi, University of Modena e Reggio Emilia
Gamma-retroviral hotspots integration in human primary keratinocytes: potential implication for clinical application
11:00-13:00
Lomond Suite
Parallel 7c
EMA Committee for Advanced Therapies - Regulatory Aspects of ATMPs
Chairs: Martina Schuessler Lenz, PEI & Ilona Reischl, AGES - MEA
INV57: Martina Schuessler-Lenz, PEI Langen
Regulatory and scientific aspects of ATMPs-– an update from the Committee for Advanced Therapies
INV58: Barbara Bonamassa, AIFA, Rome
Regulatory aspects of gene-editing based ATMPs
INV55: Claire Beuneu, AFMPS, Brussels
Current status of the ICH S12 guideline on biodistribution studies for gene therapies
INV56: Carla Herberts, CBG Meb, Amsterdam
Regulatory considerations on integrational events of recombinant AAV-based gene therapy
INV59: Ilona Reischl, EMA Amsterdam
Hot topics for ATMP developers
Round table discussion
13:00-14:30
Break
13:30-14:15
Fintry
Lunchtime Symposium
The ERC’s programme and
widening European participation:
What’s on the horizon?
Chair: Janka Mátrai, ERC
Join our ERC session to get funding updates and advice from experts. Participate in the round table discussion and ask your questions on ERC’s newest funding opportunities and actions towards widening European participation and closing the currently existing success gap. The session is organised and chaired by Janka Mátrai, and kindly supported by two distinguished guest scientists, Joanna Madzio and Zoltán Ivics
13:30 -14:15
13:30 -14:15
14:30-17:00
Pentland Suite
Plenary 5
Novel approaches on cell and gene therapy
Chairs: Hildegard Büning, Hannover Medical School / Juan Bueren, Ciemat Madrid / Rafael Yáñez-Muñoz, Royal Holloway University of London
INV66: Luca Biasco, University College London/Sana Biotechnology
Novel cellular targets and delivery strategies for hematopoietic stem/progenitor cell gene therapy
INV67: Mathias Vormehr, BioNTech
mRNA vaccines for the treatment of cancer
INV68: Bobby Gaspar, Orchard Therapeutics
Ending the devastation caused by severe genetic diseases through HSC gene therapy and newborn screening: a case study in metachromatic leukodystrophy
INV69: Claire Booth, University College London & INV70: Alesandro Aiuti, SR-Tiget, Milan
Access to gene therapies for rare diseases - it's time to act
Rafael Yáñez-Muñoz, Royal Holloway University of London
BSGCT Fairbairn Award
Hildegard Büning, Hannover Medical School
Closing words
17:00
Closing drinks
