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 | Discussion / Q&A |
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 | Discussion / Q&A |
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 | Discussion / Q&A |
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 |
Agree next steps? | |
19:20 | Wrap up and Prize Giving – Chairman of Cystinosis Ireland – Mr Mick Swift |