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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

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

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

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

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   

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

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

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

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

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 

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

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