Tuesday, 21 January 2020
The second day of the Congress, open to Industry and Standard ticket holders, focuses on the latest research and partnership working between charities, regulators, and industry. For any queries related to the programme, please email programme@ebworldcongress.org. Please note: The Congress programme is updated monthly and the current version is, therefore, subject to change.Programme overview
08:30 | WELCOME
Chairs: Jemima Mellerio & Jouni Uitto
Chairs: Jemima Mellerio & Jouni Uitto
From theory to practice – translational medicine into clinical trials
Jemima Mellerio
Jemima Mellerio
08:40 | CELL MANIPULATION & THERAPIES
Chair: John McGrath
Chair: John McGrath
Combining approaches – BMT & systemic treatment
Jakub Tolar Fibroblast gene therapy
Su Lwin HMGB1 peptide
Katsuto Tamai State of the art iPSCs
Dennis Roop Panel discussion Oral presentations 115 - Joanna Jackow
Efficient genome editing for correction of recessive dystrophic epidermolysis bullosa in iPS cells using CRISPR/Cas9 RiboNucleoProtein complexes 7 - Martin Barbier
Self-assembled skin substitutes and retroviral gene therapy for the permanent treatment of recessive dystrophic epidermolysis bullosa
Jakub Tolar Fibroblast gene therapy
Su Lwin HMGB1 peptide
Katsuto Tamai State of the art iPSCs
Dennis Roop Panel discussion Oral presentations 115 - Joanna Jackow
Efficient genome editing for correction of recessive dystrophic epidermolysis bullosa in iPS cells using CRISPR/Cas9 RiboNucleoProtein complexes 7 - Martin Barbier
Self-assembled skin substitutes and retroviral gene therapy for the permanent treatment of recessive dystrophic epidermolysis bullosa
10:25 | COFFEE BREAK
10:55 | GENE MANIPULATIONS & THERAPIES
Chair: Jakub Tolar
Chair: Jakub Tolar
State of the art gene editing
Fernando Larcher EBGRAFT and advances in skin grafting using improved vectors
Alain Hovnanian Hologene projects
Laura de Rosa Next generation sequencing applications for mutation detection in EB
Jouni Uitto Gene therapy for RDEB (ex-vivo vs in-vivo)
Peter Marinkovich Exon skipping for RDEB
Peter van den Akker COL17A1 editing using CRISPR/Cas9
Ulrich Koller Non-viral gene therapy
Lara Cutlar Panel discussion Oral presentations 81 - Jose Bonafont Arago
CRISPR/Cas9-based gene editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa 86 - Hiroyuki Morisaka
Possible application of broad and unidirectional genome editing using the novel CRISPR-Cas3 system for autosomal dominant Epidermolysis Bullosa
Fernando Larcher EBGRAFT and advances in skin grafting using improved vectors
Alain Hovnanian Hologene projects
Laura de Rosa Next generation sequencing applications for mutation detection in EB
Jouni Uitto Gene therapy for RDEB (ex-vivo vs in-vivo)
Peter Marinkovich Exon skipping for RDEB
Peter van den Akker COL17A1 editing using CRISPR/Cas9
Ulrich Koller Non-viral gene therapy
Lara Cutlar Panel discussion Oral presentations 81 - Jose Bonafont Arago
CRISPR/Cas9-based gene editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa 86 - Hiroyuki Morisaka
Possible application of broad and unidirectional genome editing using the novel CRISPR-Cas3 system for autosomal dominant Epidermolysis Bullosa
13:25 | LUNCH
14:25 | CLINICAL TRIALS & RESEARCH PROGRAMMES (includes tea break)
Chair: Johann Bauer
Chair: Johann Bauer
Introduction – clinical trial design
Johann Bauer Large wounds: an update on natural history data and EB101
Jean Tang Recombinant collagen 7: a systemic approach to RDEB therapy
Theresa Podrebarac A tale of two therapeutic approaches - an update on clinical studies in diacerein and FCX-007
Mary Spellman B-VEC off the shelf topical gene therapy for DEB
Suma Krishnan ABCB5+ mesenchymal stem cells for the treatment of recessive dystrophic epidermolysis bullosa – from bench to bedside
Kathrin Dieter Questions 16:10 | TEA BREAK Partnerships – charities, regulators, and industry
Brett Kopelan Partnership Approach to research funding
Sharmila Collins
Johann Bauer Large wounds: an update on natural history data and EB101
Jean Tang Recombinant collagen 7: a systemic approach to RDEB therapy
Theresa Podrebarac A tale of two therapeutic approaches - an update on clinical studies in diacerein and FCX-007
Mary Spellman B-VEC off the shelf topical gene therapy for DEB
Suma Krishnan ABCB5+ mesenchymal stem cells for the treatment of recessive dystrophic epidermolysis bullosa – from bench to bedside
Kathrin Dieter Questions 16:10 | TEA BREAK Partnerships – charities, regulators, and industry
Brett Kopelan Partnership Approach to research funding
Sharmila Collins
17:00 | CURRENT DILEMMAS - A CONGRESS DEBATE (Measurement & research)
Chair: Jouni Uitto
Chair: Jouni Uitto
EB relevant endpoints
Dedee Murrell Quality of life measures
Elena Pope Regulatory perspectives and challenges
Brett Kopelan Planning for clinical translation
Catriona Crombie Innovative therapeutic development
Mauro Peretti Open debate – how to direct and support the development of meaningful outcome measures to ensure a positive outcome; working together – charities, regulators, and industry – where are the opportunities and gaps
Dedee Murrell Quality of life measures
Elena Pope Regulatory perspectives and challenges
Brett Kopelan Planning for clinical translation
Catriona Crombie Innovative therapeutic development
Mauro Peretti Open debate – how to direct and support the development of meaningful outcome measures to ensure a positive outcome; working together – charities, regulators, and industry – where are the opportunities and gaps
17:45 | CLOSE