Dublin Cystinosis Workshop 2026

The annual 12th Dublin Cystinosis Workshop took place online on Tuesday 10^th February. This year’s theme was to showcase the breadth of innovative research that Cystinosis Ireland funds, worldwide. The day featured presentations from Cystinosis Ireland–funded researchers, highlighting significant advancements in cystinosis treatment and disease understanding. Session 1 opened with an introduction by Anne-Marie O'Dowd, Chair of Cystinosis Ireland’s Research Committee, who highlighted the organisation's investment in research worth 3.8 million euros. Dr Reza Sajadi, the chair of the scientific committee, introduced the agenda.

Dr Jennifer Hollywood opened the presentations showing new findings from rat models demonstrating immune‑driven fibrotic remodelling in cystinosis‑affected kidneys. Her second presentation outlined promising results from combination therapy for nephropathic cystinosis in a new cystinotic rat model. Dr Stephanie Myers followed with an update on the development of fluorescent probes designed to detect methanethiol, a key metabolic product of cysteamine. Dr Francesco Bellomo then discussed progress in regenerating a knockout mouse model to support deeper study of cystinosis pathophysiology. Prof Elena Levtchenko presented exciting progress on mRNA‑based therapies for cystinosis.

Session 1 concluded with an engaging Q&A that explored topics such as muscle cysteine accumulation, therapeutic targeting, and detection methodologies, while also addressing both the challenges and potential pathways for future treatment innovation.

Following the Q&A, presenters hosted breakout rooms where attendees could meet directly with the speaker of their choice for further discussion.

Session 2 began with an update from Prof Roos Masereeuw on the EU‑funded MSCA Joint Doctoral Network ORGESTRA, a Marie Skłodowska‑Curie training programme supporting 13 PhD candidates advancing organoid technologies for disease modelling, drug discovery, and therapy development for rare diseases, including cystic fibrosis and cystinosis. Cystinosis Ireland is an associate partner on this programme.

Dr Brendan Keating then delivered a session on human genome sequencing and its potential for future precision‑medicine strategies. This was followed by Dr Anuj Chauhan’s update on his HRCI-HRB JFS project, which focuses on sustained delivery of cysteamine prodrugs using nano barrier contact lenses. Dr Sastika Sur, presenting on behalf of Prof. Minnie Sarwal, provided an overview of their work on nephropathic cystinosis, integrating mechanistic insights with nano‑delivery approaches.

Dr Tracey McCauley, Research Manager at Cystinosis Ireland, presented an overview of the organisation’s funding schemes.

The keynote lecture was delivered by Dr. Jaime Cheah, who provided an in‑depth look at the drug‑repurposing library at the Broad Institute, outlining its development, composition, and open‑access model.

The session—and the 2026 Dublin Cystinosis Workshop—closed with remarks from Anne Marie O’Dowd.

The Scientific Committee for the 2026 Dublin Cystinosis Workshop played an essential role in shaping this year’s programme. Through their scientific oversight, careful review, and guidance, they ensured that the workshop delivered a high‑quality, relevant, and forward‑looking agenda for all participants.

The Research Committee of Cystinosis Ireland continues to underpin every aspect of the organisation’s research strategy. Their expertise, thoughtful evaluation, and long‑standing commitment ensure that all funded projects remain rigorous, strategic, and firmly aligned with the needs and priorities of the cystinosis community.

Cystinosis Ireland also acknowledges the dedication of its funded researchers, whose contributions extend far beyond scientific excellence. Their ongoing engagement with patients and families, openness to collaboration, and commitment to addressing community‑identified research priorities play a vital role in advancing the field.