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Friday 14 October



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



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.


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



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

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Parallel 7b

Nanoparticles and


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

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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 BonamassaAIFA, 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

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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

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13:30 -14:15

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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

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Closing drinks 

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