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 subject to change.

Programme overview

08:30 | WELCOME
Chair: Jemima Mellerio
From theory to practice – translational medicine into clinical trials
Jemima Mellerio
08:40 | CELL MANIPULATION & THERAPIES
Chair: John McGrath
Combining approaches – BMT & systemic treatment
Jakub Tolar

Dutch experience – stem cells
Vamsi Yenamandra

Cell therapy – MSCs/Lenticol
Su Lwin

HMGB1 peptide
Katsuto Tamai

State of the art iPSCs
Dennis Roop

Oral presentations

Panel discussion
10:25 | COFFEE BREAK
10:55 | GENE MANIPULATIONS & THERAPIES
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 approaches for mutation detection
TBD

Gene therapy for RDEB (ex-vivo vs in-vivo)
Peter Marinkovich

Exon skipping for RDEB
Peter van den Akker

CRISPR in Col17
Ulrich Koller (EB Haus)

Non-viral gene therapy
Lara Cutlar

Oral presentations

Panel discussion
13:20 | LUNCH
14:20 | CLINICAL TRIALS
Chair: Martin Laimer
Introduction – clinical trial design
Martin Laimer

Industry presentations

Panel discussion
15:50 | TEA BREAK
16:20 | WORKING TOGETHER
EB partnerships, charities, regulators, and industry
Brett Kopelan
16:30 | CURRENT DILEMMAS - MEASUREMENT & RESEARCH (panel debate)
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 (LifeArc)

Innovative therapeutic development
Mauro Peretti

Panel 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
17:15 | CLOSE
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