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Wednesday 12 October



Pentland and Sidlaw

Parallel 2a

Vaccine technologies & infectious diseases


Chairs: Evangelia Yannaki, G.Papanikolaou Hospital, Thessaloniki / James Miskin, Oxford BioMedica

INV10: Jerome Custers, Janssen, Johnson & Johnson

INV11: Darin Edwards, Moderna Therapeutics

Advancements in mRNA Science and Technology enabled a platform for rapid vaccine development against pandemic



INV12: Ami Patel, The Wistar Institute, Philadelphia

From Pathogen to Protection: Engineering synthetic DNA-encoded vaccines and immunotherapies against emerging and pandemic viral pathogens

OR14: Derek Jantz, Precision Biosciences Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate Hepatitis B virus in vivo


OR15: Allan Scarpitta, University of Rouen

AAV-mediated induction of immunogenic cell death to promote anti-tumor immune responses


P500: Frederick Rothemejer, Aarhus University

HIV-resistant anti-HIV CAR T cells as a functional cure


P504: Jacqueline Doms, ART-TG, Inserm France

A non-viral CRISPR/Cas9 gene-editing approach to express anti-HIV broadly-neutralizing antibodies in human B cells for HIV immunotherapy



Parallel 2b

Disease modelling in advanced therapies

Chairs: Gerry McLachlan, University of Edinburgh / GIorgia Sanitlli, UCL, London

INV13: Patapia Zafeiriou, University Medical Center Goettingen

Human bioengineered neuronal organoids as preclinical models for gene therapy

INV14: Meritxell Huch, MPI-CBG Dresden

Exploiting organoids as disease models

OR16: Ranjita Devi Moirangthem, Institut Imagine, Paris

Ex Vivo Immunotherapeutic NK Cell Production From Cord Blood Or Mobilized Peripheral Blood CD34+ Cells Using Notch Ligand Delta-Like 4 Culture System


OR17: Els Verhoeyen, University of Nice

Nanoblades allow high-level genome editing in organoids,


OR18: Myriam Lemmens, Novartis

Identification of marker genes to monitor residual iPSCs in iPSC-derived products


OR19: Liam Kempthorne, UCL London

Utilising CRISPR-Cas13 systems to target frontotemporal dementia and amyotrophic lateral sclerosis-causing C9orf72 repeat expansion-containing RNA


P350: Caterina Gomes, Ibet Lisboa

Human stem-cell based models to study innate immunity and neuroinflammation in the central nervous system


Lomond Suite

Parallel 2c

Metabolic diseases 

Chairs: Fulvio Mavilio, Orchard Therapeutics / Fatima Bosch, UAB Barcelona

INV15: Andrea Annoni, SR-Tiget, Milan

Inverse vaccination strategies for the induction of Ag-specific immune tolerance in autoimmune diseases

INV16: Paolo Martini, Moderna Therapeutics

Messenger RNA therapy is a platform therapeutic for the treatment of Rare
Genetic Disorders

OR20: Tarekegn Geberhiwot, University of Birmingham

Final safety and efficacy of a phase 1/2 trial of DTX301 in adults with late-onset ornithine transcarbamylase deficiency (OTCD)


OR21: P. Deegan, Addenbrooke’s Hospital, Cambridge

Preliminary results of STAAR, a Phase I/II study of isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease and long-term follow-up 


OR22: Loukia Touramanidou, UCL London

In vivo lentiviral gene therapy restores ureagenesis in the urea cycle defect argininosuccinic aciduria


OR23: Randy Chandler, National Human Genome Research Institute

Gene Therapy to Treat Methylmalonic and Propionic Acidemia Using the Novel Adeno-associated Viral Capsid 44.9


P674: Estera Rintz, University of Gdansk

Adeno-associated virus vector combination gene therapy with C-type natriuretic peptide and GALNS enzyme in Mucopolysaccharidosis IVA mouse model


P537: Bethan Critchley, UCL London

Targeting the blood brain barrier for haematopoietic stem cell gene therapy in neurological lysosomal storage disorders


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Pentland Suite

Plenary 2

Clinical Trials


Chairs: Axel Schambach, Hannover Medical School / Giuliana Ferrari, SR-Tiget, Milan

INV17: Peter Marinkovich, Stanford University School of Medicine

Topical gene therapy for dystrophic epidermolysis bullosa, results of a phase 3 trial

INV18: Bernhard Gentner, SR-Tiget, Milan

Genetically-modified hematopoietic stem cells as a one-time, systemic treatment for non-hematologic disorders.


INV19: Nicola Brunetti-Pierri, Tigem, Naples

Liver-Directed Gene Therapy for Mucopolysaccharidosis Type VI


INV20: Don Kohn, University of California, Los Angeles

Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I) 

INV21: Pratima Chowdary, UCL, London

A novel adeno-associated virus (AAV) gene therapy (FLT180a) achieves normal FIX activity levels in severe Hemophilia B (HB) patients (B-AMAZE study)

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Lunchtime sponsor


Chair:Jude Samulski, AskBio


Cesar Trigueros, Viralgen, San Sebastian

A Platform Approach For Adeno-Associated Virus: Manufacturing To Support Gene Therapy Product.

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Lunchtime sponsor


Chairs: Roland Leathers, Strategic Alliances Manager, ThermoFisher Scientific

Helena Meyer-Berg, AAV Platform Manager Project Developer
Cell & Gene Therapy Sirion Biotech


Toke JostIsaken, Senior Scientist, RNA & Gene Therapies, Global
Research Technologies, Novo Nordisk A/S

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Lunchtime sponsor



Philippe MoyenPTC Therapeutics

PTC Therapeutics: partners for pDNA and clinical AAV Vector manufacturing

Dr Vincent D’HardemareService de neurochirurgie adultes – Hospital Fondation Rothschild

Intraputaminal gene therapy for AADC deficiency: Surgery and outcomes

Philippe Moyen and Dr Vincent D’Hardemare

Panel discussion

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Pentland / Sidlaw

Parallel 3a

CNS and sensory diseases I 

Chairs: Alberto Auricchio, Tigem, Naples / Rajvinder Karda, UCL London

INV26: Krystof Bankiewicz, University College London

MR-guided midbrain administration of AAV2AADC in patients with AADC deficiency results in clinical improvement

INV27: Nicole Deglon, Centre Hospitalier Universitaire Vaudois (CHUV), Lausanne

Genetic engineering to the rescue of Huntington’s disease

OR30: Amy Geard, UCL London

AAV9-mediated gene therapy in a knock-in mouse model of infantile neuroaxonal dystrophy


OR31: M Kaplitt, Weill Cornell Medical College, NYC

Gene therapy in APOE4 homozygote Alzheimer’s disease – interim data


OR32: Kevin Nash, University of South Florida

Reelin Gene Therapy for Fragile X Syndrome


P184: Sergi Verdes, UAB Baracelona

Gene therapy for ALS by specifically overexpressing a pleiotropic chronokine, secreted α-Klotho, in skeletal muscles


P185: Kathrin Meyer, Nationwide Children’s Hospital

A Novel AAV Gene Therapy for Rett Syndrome through Reactivation of the Silent X Chromosome

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Parallel 3b

Advances in Haemophilia A

Chairs: Christos Georgiadis, UCL London / HIldegund Ertl, The Wistar Institute, Philadelphia  

INV22: Kevin Eggan, BioMarin


INV23: Antonia Follenzi, Univesita del Piemonte Orientale, Novarra

Targeting FVIII  expression to liver endothelium 

OR24: Felix Lansig, Dresden University
Fusion of Designer recombinases for efficient and specific correction of a Factor VIII genomic inversion


OR25: Federica Esposito, Tigem, Naples

AAV-mediated homology-independent targeted integration leads to sustained secretion of therapeutic proteins from new-born liver.


OR26: Sara Deola, Sidra Medicine

Assessment of FVIII in the hematopoietic system: UM171-expanded CD31+ monocytes show the highest GT potential to correct HA

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Lomond Suite

Parallel 3c

Advanced therapies for solid tumors  

Chairs: Cristina Fillat, IDIBAPS, Barcelona / Alan Parker, Cardiff University

INV24: Marta Alonso, University of Navarra

Virotherapy for pediatric brain tumors: more than a hope

INV25: Guy Ungerechts, NCT/DKFZ, Heidelberg

Immunovirotherapy – Clinical Translation

OR27: Thomas Kerzel, SR Tiget, Milan

IFNalpha by in vivo-engineered macrophages abates liver metastases and triggers counter regulatory responses limiting treatment efficacy


OR28: Alicia Teijeira Crespo, University of Cardiff

Development of a tumour selective precision immunovirotherapy expressing immune checkpoint inhibitors targeting LAG3


OR29: Alessia Potenza, SR Tiget, Milan

Harnessing CD39 for the treatment of colorectal cancer and liver metastases by engineered T cells


P376: Ryan Murray, Northeastern University

Multiplex base editing protects allogeneic solid tumor targeting CAR-T cells from inhibition by extracellular adenosine in the tumor microenvironment


P382: Owen Moon, InstilBio

Antitumor activity of T cells expressing a novel anti-folate receptor alpha (FRα) costimulatory antigen receptor (CoStAR) in a human xenograft murine solid tumor model and implications for in-human studies

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Pentland Suite

Plenary 3

AAV safety

Chairs: Hildegard Büning, Hannover Medical School / Alberto Auricchio, Tigem, Naples

INV28: Terry Flotte, University of Massachusetts

Overview of Clinical Gene Therapy with AAV from the Safety Perspective


INV29: Denise Sabatino, Children's Hospital of Philadelphia

Current understanding of AAV integration and the potential risk of AAV-associated insertional mutagenesis

INV30: Juliette Hordeaux, University of Pennsylvania

AAV-related toxicities in nonhuman primates

Round Table 

Carla Herberts, Hildegard Büning, Terry Flotte, Denise Sabatino, Juliette Hordeaux, Declan Noone

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Poster session 1 ( Poster with an ODD number )

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