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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

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
From theory to practice – translational medicine into clinical trials
Jemima Mellerio
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
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
13:25 | LUNCH
14:25 | CLINICAL TRIALS & RESEARCH PROGRAMMES (includes tea break)
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


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
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
17:45 | CLOSE