Wednesday 12 October
07:30-08:30
Registration
08:30-10:40
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
SARS-CoV-2
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
08:30-10:40
Fintry
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
08:30-10:40
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
10:40-11:10
Break
11:10-13:15
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)
13.15-15:15
Lunch
13:45-14:45
Pentland
Lunchtime sponsor
symposium
Chair:Jude Samulski, AskBio
Cesar Trigueros, Viralgen, San Sebastian
A Platform Approach For Adeno-Associated Virus: Manufacturing To Support Gene Therapy Product.
13:45-14:45
Fintry
Lunchtime sponsor
symposium
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
14:00-15:00
Sidlaw
Lunchtime sponsor
symposium
Philippe Moyen, PTC Therapeutics
PTC Therapeutics: partners for pDNA and clinical AAV Vector manufacturing
Dr Vincent D’Hardemare, Service de neurochirurgie adultes – Hospital Fondation Rothschild
Intraputaminal gene therapy for AADC deficiency: Surgery and outcomes
Philippe Moyen and Dr Vincent D’Hardemare
Panel discussion
15:15-17:10
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
15:15-17:10
Fintry
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
15:15-17:10
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
17:10-17:40
Break
17:40-19:30
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
19:30-21:00
Poster session 1 ( Poster with an ODD number )
