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Monday 17th June



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

TL02: Exploiting Adenovirus Biology for Advancing Adeno-Associated Viral Vector Manufacturing

Weiheng Su, OxGene/WuXi

TL03: Innovation Hubs for Gene Therapies initiative in the UK

Piers Walser, NHSBT Bristol

Nelson Mandela Auditorium

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

Coffee Break


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

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 

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

Poster Session 1 and Networking

Founders Room and Seminar Room A

Tuesday 18th June



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   

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

Coffee Break



Keynote Session 2: Stem Cell Therapy
Chairs: Florian Merkle, University of Cambridge and Ellie Chilcott, University College London

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





BSGCT members are invited to attend our Annual General Meeting to hear updates on the society, our committee and our activities.

Nelson Mandela Auditorium


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


Coffee Break



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

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

Poster Session 2 and Networking

Founders Room and Seminar Room A

Conference Dinner

After-Dinner Speaker

Catherine Green, University of Oxford

Keble College

Wednesday 19th June





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 

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


Coffee Break



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

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