The Dublin Cystinosis Workshop is an interactive forum for researchers to share new knowledge, spark new ideas and explore new research approaches aimed at better understanding and treating the ultra-rare disease of cystinosis

THIS IS A CLOSED WORKSHOP

PARTICIPATION IS BY INVITATION ONLY OR VIA THE CALL FOR ABSTRACTS

You can find the meeting agenda here.

THE 7TH ANNUAL DUBLIN CYSTINOSIS WORKSHOP 2021 WILL BE HELD ON 20 MAY 2021

As in other years, the Virtual 7th Annual Dublin Cystinosis Workshop 2020 is a scientifically focused workshop that brings together internationally recognised researchers and clinicians from Ireland, the UK, Europe, North America and New Zealand to share the most recent advances in their respective research endeavours and to look at new ways to develop our understanding of cystinosis as a disease and its treatment.

The Dublin Cystinosis Workshop is the only such scientific workshop in Europe focused on cystinosis.

The aim of the workshop is to share new research developments in the area of cystinosis and to stimulate discussions and collaborations that can convert new ideas into real research that moves the cystinosis research field forward.

Topics presented at the workshop will cover the spectrum of basic through to translational research.

In particular, researchers who have been funded by or have applied to Cystinosis Ireland for funding are encouraged to present their research at the workshop.

Early stage researchers and PhD students are strongly encouraged to present their research at the workshop.

As it is a closed meeting (no industry) and participation is by invitation only, researchers are free to present their latest scientific work to colleagues spanning a wide range of cross-cutting disciplines from basic through to translational research.

This year, the workshop will take the format of include presentations from a number of keynote speakers and a series of shorter research communications presenting latest research results and new research directions in the cystinosis research world.

This year, we will also have a particular focus on clinical trials being conducted in cystinosis worldwide and we also hope to inform researchers about key infrastructures here in Ireland that make it an attractive location to host a clinical trial.

We look forward to seeing you virtually on the 20th May.

FOR MORE INFORMATION ON THE 7TH ANNUAL DUBLIN CYSTINOSIS WORKSHOP INCLUDING THE WORKSHOP AGENDA – CLICK HERE

Please note, attendance at the Dublin Cystinosis Workshop is by invitation only.  If you would like to attend, please email [email protected] for more details.

7th Annual Dublin Cystinosis Workshop, 20th May 2021

Cystinosis Ireland is proud to announce the 7th Annual Dublin Cystinosis Workshop will take place online, on Thursday 20 May 2021 from 11:45 to 19:30 (Irish Local Time).

All times below are Dublin, Ireland local time.  If you would like to attend the Dublin Cystinosis Workshop, please email [email protected].

11:45 Welcome to delegates – Session Chair: Prof Elena Levtchenko
12:00 Keynote Scientific speaker – Professor David Sabatini, Professor of Biology, Whitehead Institute, MIT, USA – MFSD21 transports cysteine into lysosomes and melanosomes
12:30 Discussion / Q&A
12:40 Cystinosis Research – New Research Results and Novel and Exploratory Research Directions – Short Communiques – Session Chair: Dr Patrick Harrison
12:40 Dr Jennifer Hollywood – University of Auckland, New Zealand – MFSD12 a candidate therapeutic target for a new class of cystinosis drugs?
12:50 Pang Yuk Cheung – University of Auckland, New Zealand – Optimisation of oral cysteamine dosing in Ctns knockout rats
13:00 Tjessa Bondue – KU Leuven, Belgium – The potential of CTNS-mRNA loaded nanoparticles to treat cystinosis
13:10 Elena Sendino Garví – Utrecht University, The Netherlands – Generation of a novel bioengineered 3-dimensional model of renal cystinosis
13:20 Louise Medaer – KU Leuven Belgium – Employing isogenic cell models to study the interactome of cystinosin and the underlying mechanism of cystinosis myopathy
13:30

14:00

Discussion / Q&A

Break

14:15 Keynote Scientific Speaker – Professor John Foxe –  University of Rochester School of Medicine and Dentistry – Exploring the Neurophysiology of Cystinosis.  Session Chair: Dr Jennifer Hollywood
14:45 Discussion / Q&A
14:55 Cystinosis Research – New Research Results and Novel and Exploratory Research Directions – Short Communiques – Session Chair: Dr Jennifer Hollywood
14:55 Dr Valeria Graceffa – Institute of Technology Sligo, Ireland – Fibrin hydrogels releasing the CTNS gene for the treatment ocular cystinosis 
15:05 Dr Roisin O’Flaherty – Maynooth University, Ireland – Serum IgG Glycosylation in Cystinosis
15:15 Dr Emyr Lloyd-Evans – Cardiff University, Wales – Introducing the Cardiff University Rare diseasE (CURE) network to the cystinosis community and Novel fluorescence probe approach for analysing lysosomal cystine accumulation and clearance by microscopy 
15:25 Dr Julia Hannah Quitmann – University Medical Centre, Hamburg, Germany – Development of a patient-reported outcome to measure health-related quality of life in children and adolescents with cystinosis 
15:35 Dr Swastika Sur – University of California, San Francisco, USA – Loss of Renal Epithelial Cell-Specific V-ATPase Subunit Restricts Mitochondrial Function and Autophagosome-Lysosome Clearance by Perturbing Intracellular Acidic pH in Nephropathic Cystinosis 
15:45

16:15

Discussion / Q&A

Break

16:45 Ireland as a cystinosis clinical trial hub – Session Chair: Prof Atif Awan
16:45 Dr Jennifer Ralph James – Head of the National Office for Research Ethics Committees, Ireland – Irish Doors are Open for Clinical Trial and Medical Device Research
16:55 Dr Geraldine O’Dea – Health Products Regulatory Agency, Ireland – The HPRA and regulation of clinical trials in Ireland
17:05 Dr Cormac McCarthy / Dr Rachel Crowley – University College Dublin, Ireland – A Rare Disease Clinical Trials Network in Ireland
17:15 Professor Francesco Emma – Worldwide Cystinosis Registry … A key infrastructure for cystinosis clinical trials
17:30

17:45

Discussion / Q&A

Break

18:00 Snapshots of clinical trials in the cystinosis world – Session Chair: Prof Elena Levtchenko
18:00 Professor Herbie Newell C.B.E. – University of Sunderland, UK – An update on the development of the cysteamine prodrug CF10
18:10 Professor Paul Goodyer MD – McGill University, Canada – Update on the development of a drug therapy for cystinosis nonsense
18:20 Professor Anuj Chauhan – Colorado School of Mines, CO, USA – Cystinosis therapy by contact lenses
18:30 Professor Stephanie Cherqui – UCSD, USA – Hematopoietic stem cell gene therapy for cystinosis: updated results from a phase I/II clinical trial
18:40 Cécile L. Berends – Centre for Human Drug Research, Leiden, The Netherlands – A novel sustained‐release cysteamine bitartrate formulation for the treatment of cystinosis: Pharmacokinetics and safety in healthy male volunteers
18:50 Discussion / Q&A
19:05 Voting on best presentation (short communiques)
19:10 Professor Dr Elena Levtchenko – KU Leuven, Belgium – Heads up on next EU Rare Disease programme 
19:20 Wrap up and Prize Giving – Chairman of Cystinosis Ireland – Mr Mick Swift 

Conference Chairs:

Professor Elena Levtchenko

Dr Patrick Harrison

Professor Atif Awan

Dr Jennifer Hollywood

Speakers:

Professor David Sabatini – Professor of Biology, Whitehead Institute, MIT, USA
Dr Jennifer Hollywood – University of Auckland, NZ
Pang Yuk Cheung – University of Auckland, NZ
Tjessa Bondue – KU Leuven, BE
Elena Sendino Garví – Utrecht University, NL
Louise Medaer – KU Leuven BE
Professor John Foxe –  University of Rochester School of Medicine and Dentistry, USA
Dr Valeria Graceffa – IT Sligo, Ireland
Dr Roisin O’Flaherty – Maynooth University, Ireland
Dr Emyr Lloyd-Evans – Cardiff University, Wales
Dr Julia Hannah Quitmann – University Medical Centre, Hamburg, Germany
Dr Swastika Sur – UCSF, USA
Dr Jennifer Ralph James – Head of the National Office for Research Ethics Committees, Ireland
Dr Geraldine O’Dea Health – Products Regulatory Agency, Ireland
Dr Cormac McCarthy – UCD, Ireland
Professor Francesco Emma – Ospedale Pediatrico Bambino Gesù, Rome, Italy
Professor Herbie Newell C.B.E. – University of Sunderland, UK
Professor Paul Goodyer MD – McGill University, Canada
Professor Anuj Chauhan – Colorado School of Mines, CO, USA
Professor Stephanie Cherqui – UCSD, USA
Cécile L. Berends – Centre for Human Drug Research, Leiden, The Netherlands

Virtual 7th Annual Dublin Cystinosis Workshop 2021 Scientific Steering Committee

  • Co-Chairperson – Professor Elena Levtchenko, Paediatric Nephrologist, UZ Leuven
  • Co-Chairperson and Host Country Medical Organiser Paediatric – Professor Atif Awan, Consultant Paediatric Nephrologist, Temple Street Children’s University Hospital, Dublin
  • Dr Paddy Harrison – Senior lecturer, Department of Physiology, University College Cork
  • Dr Jennifer Hollywood – University of Auckland, NZ
  • Dr Thomas J McDonald – Director, Drug Product EMEA/APAC External Manufacturing, Bristol Myers Squibb – Cystinosis Ireland Board Member
  • Dr Achim Treumann – Director, Analytical Formulation Sciences and Site Head at KBI Biopharma BV – Cystinosis Ireland Scientific Advisor
  • Ms Anne Marie O’Dowd – Chairperson Cystinosis Network Europe, and Board Member of Cystinosis Ireland
  • Dr Ruth Davis – Research Manager, Cystinosis Ireland
  • Ms Denise Dunne – Operations Manager, Cystinosis Ireland