Programme

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Wednesday 12 October

07:30-08:30
Registration

08:30-10:40

Parallel 2a

Vaccine technologies

Jerome Custers, Curevac

Speaker 2

 

3x selected oral presentations

2x lightning poster presentations

08:30-10:40

Parallel 2b

Disease models: iPS derived and organoids

Patapia Zafeiriou, University of Goettingen

Meritxell Huch, University of Cambridge

 

3x selected oral presentations

2x lightning poster presentations

08:30-10:40

Parallel 2c

Metabolic diseases 

Andrea Annoni, SR-Tiget, Milan

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

Paolo Martini, Moderna Therapeutics

3x selected oral presentations

2x lightning poster presentations

10:40-11:10
Break

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11:10-13:15

Plenary 2

Clinical Trials

 

Peter Marinkovich, Stanford University School of Medicine

Topical gene therapy of the skin


Bernhard Gentner, SR-Tiget, Milan

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

 

Nicola Brunetti-Pierri, Tigem, Naples

Liver-Directed Gene Therapy for Mucopolysaccharidosis Type VI

 

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) 

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13.15-15:15
Lunch

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15:15-17:10

Parallel 3a

Gene therapy of infectious diseases


Ami Patel, The Wistar Institute, Philadelphia

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

Invited Speaker

2x selected oral presentations

2x lightning poster presentations

15:15-17:10

Parallel 3b

Oncolytic viruses  

Marta Alonso, University of Navarra

Virotherapy for pediatric brain tumors: more than a hope

Guy Ungerechts, NCT/DKFZ, Heidelberg

Immunovirotherapy – Clinical Translation

2x selected oral presentations

2x lightning poster presentations

15:15-17:10

Parallel 3c

CNS and sensory I 

Krystof Bankiewicz, University College London

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

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

Genetic engineering to the rescue of Huntington’s disease

2x selected oral presentations

2x lightning poster presentation

17:10-17:40
Break

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17:40-19:30

Plenary 3

AAV safety

Terry Flotte, University of Massachusetts

Overview of Clinical Gene Therapy with AAV from the Safety Perspective

 

Denise Sabatino, Children's Hospital of Philadelphia

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

Juliette Hordeaux, University of Pennsylvania

AAV-related toxicities in nonhuman primates

Round Table 

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19:30-21:00
Poster session 1

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