Monday 17th June
12:30-15:00
Registration
Foyer
13:30-15:00
Process Development/Technological Highlights
Chairs: James Miskin, Independent and Steven Howe, Resolution Therapeutics
TL01: Lentiviral vector process development and GMP manufacturing
Carol Knevelman, Oxford Biomedica
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TL02: Exploiting Adenovirus Biology for Advancing Adeno-Associated Viral Vector Manufacturing
Weiheng Su, OxGene/WuXi
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TL03: Innovation Hubs for Gene Therapies initiative in the UK
Piers Walser, NHSBT Bristol
Nelson Mandela Auditorium
15:00-16:45
Opening Session: Genome Editing / Blood
Chairs: Steve Hyde, University of Oxford and Rafael J. Yáñez-Muñoz, Royal Holloway University of London
Welcome and opening remarks
Steve Hyde, Chair of Local Organising Committee and Rafael J. Yáñez-Muñoz, BSGCT President
INV01: Gene Editing for Inborn Errors of Immunity
Emma Morris, UCL Institute of Immunity & Transplantation
INV02: Genome editing for inherited anaemias
James Davies, University of Oxford
OR01: Traceless delivery of prime editor ribonucleoprotein (RNP) complexes in engineered nanoparticles
Jakob Hansen Haldrup, University of Oxford
OR02: Developing a universal haematopoietic stem cell gene editing therapy for XIAP deficiency
Annelotte Mudde, University College London
Nelson Mandela Auditorium
16:45-17:15
Coffee Break
Foyer
17:15-19:00
Keynote Session 1 and Fairbairn Presentations
Chairs: Gerry McLachlan, University of Edinburgh and Stephanie Jones, University of Oxford
KEY1: Exploring the Molecular Basis for Variation in AAV Transduction: Guiding the Selection of Optimal AAV Capsids for Clinical Trials
Mark Kay, Stanford University School of Medicine, USA
FB01: Rescue of lethal SP-B deficiency in a murine model using lentiviral vector-mediated gene therapy
Kamran Miah, University of Oxford
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FB02: Generation of a de novo intronic junction in the DMD gene through CRISPR/Cas genome editing as a potential therapy for a high proportion of Duchenne Muscular Dystrophy patients
Rebeca Gil Garzon, Royal Holloway University of London
FB03: Liver-directed lentiviral gene therapy is safe and curative in Argininosuccinic aciduria
Loukia Touramanidou, Great Ormond Street Institute of Child Health, University College London
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FB04: Safety and efficacy analysis of in vivo lentiviral gene therapy for ARC syndrome
Andrei Claudiu Cozmescu, Great Ormond Street Institute of Child Health, University College London / NIHR Great Ormond Street Hospital Biomedical Research Centre, London
Nelson Mandela Auditorium
19:00-20:00
Poster Session 1 and Networking
Founders Room and Seminar Room A
Tuesday 18th June
08.00-08:30
Registration
Foyer
08:30-10:00
Oligonucleotide and RNA
Chairs: Thomas Roberts, University of Oxford and Rebeca Gil Garzon, University College London
INV03: Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities
Aurelie Goyenvalle, Laboratoire Biothérapies des maladies neuromusculaires - Inserm
INV04: Applications of small activating RNAs in Medicine
Bríd Ryan, MiNA Therapeutics
OR03: Administration of mRNA alleviates disease biomarkers in a mouse model of pulmonary alveolar proteinosis (PAP)
Houze Zhang, Imperial College London/ UK Respiratory Gene Therapy Consortium
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OR04: Synthetic modified mRNA administration in a first in-human ex-situ heart failure model validates its use as a novel, effective and safe method of evaluating drug exposure and efficacy
John Louca, University of Cambridge
Nelson Mandela Auditorium
10.00-10.30
Coffee Break
Foyer
10.30-12.00
Keynote Session 2: Stem Cell Therapy
Chairs: Florian Merkle, University of Cambridge and Ellie Chilcott, University College London
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KEY2: Harnessing fibroblast heterogeneity for skin regeneration
Fiona Watt, EMBO, Germany
INV05: Culture-acquired genetic changes in human pluripotent stem cells: implications for basic research and regenerative medicine
Ivana Barbaric, University of Sheffield
OR05: Molecular characterisation of a novel knock-out mouse model in the context of MPSIVA ex-vivo gene therapy development
Margherita Berti, Università Vita-Salute San Raffaele / San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET)
OR06: Towards a scalable, closed and automated platform for the production of cost-efficient allogeneic cell therapies: showcase of an exemplar ink process
Alexandru Robert Podovei, Cell and Gene Therapy Catapult
Nelson Mandela Auditorium
12.00-13.30
Lunch
Foyer
12.30-13.15
BSGCT AGM
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BSGCT members are invited to attend our Annual General Meeting to hear updates on the society, our committee and our activities.
Nelson Mandela Auditorium
13:30-15:30
Ocular & CNS
Chairs: Carly Bliss, Cardiff University and Amy Geard, University College London
INV06: Gene therapy for an inherited childhood parkinsonism - dopamine transporter deficiency syndrome
Simon Waddington, University College London
INV07: Genetic therapies for retinal disease
Jasmina Kapetanovic, University of Oxford
INV08: A Self-regulating Gene Therapy for Rett Syndrome
Ralph Hector, University of Edinburgh
OR07: IKC159V: A Next Generation Bicistronic Gene Therapy for the Treatment of Geographic Atrophy
Katie Binley, Ikarovec
OR08: Living with Phelan McDermid syndrome
Katie Greed, Phelan-McDermid Syndrome Foundation (UK)
Nelson Mandela Auditorium
15:30-16:00
Coffee Break
Foyer
16.00-17:30
Cancer
Chairs: Robert Carlisle, University of Oxford and Alicia Teijeira Crespo, Cardiff University
INV09: CAR T cell therapy in hostile solid tumours
Sophie Papa, King's College London
INV10: Development of genetically refined “precision virotherapies” suitable for systemic anti-cancer applications
Alan Parker, Cardiff University
OR09: Development of a precision immunovirotherapy expressing a folate receptor α bispecific immune cell engager for treatment of ovarian cancer
Rebecca Bayliss, Cardiff University
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OR10: Single cell sequencing analysis of a CD28/CD40-based chimeric costimulatory antigen receptor (CoStAR™) activity reveals multiple functionally validated effector activities in CD4+ and CD8+ T cells
John Bridgeman, Instil Bio
Nelson Mandela Auditorium
17:30-19:00
Poster Session 2 and Networking
Founders Room and Seminar Room A
19:30-23:30
Conference Dinner
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After-Dinner Speaker
Catherine Green, University of Oxford
Keble College
Wednesday 19th June
08:30-09:00
Registration
Foyer
09:00-10:30
CV & Respiratory
Chairs: Stuart Nicklin, University of Glasgow and Marina Cerezuela, University of Oxford
INV11: Gene therapy for rare surfactant protein deficiencies
Deborah Gill, University of Oxford
INV12: Genetic therapies for inherited heart muscle disease
Hugh Watkins, University of Oxford
OR11: Toward combined cell and gene therapy for airway epidermolysis bullosa
Robert Hynds, UCL Institute of Child Health
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OR12: Non-viral GM-CSF Gene Therapy is an Effective Treatment in a Novel Murine Model of Autoimmune PAP
Claudia Ivette Juarez Molina, Imperial College London
Nelson Mandela Auditorium
10:30-11:00
Coffee Break
Foyer
11:00-12:30
Closing Session: Keynote 3 and Awards Presentations
Chairs: Steve Hyde, University of Oxford and Rafael J. Yáñez-Muñoz, Royal Holloway University of London
KEY3: Gene Therapy enabling a universal curative strategy for haemoglobinopathies
Josu De La Fuente, Imperial College, London
Awards ceremony & closing remarks
Nelson Mandela Auditorium