We extend a warm welcome to you to the Citrin Foundation Global Symposium 2025, our third international gathering dedicated to advancing research and understanding of citrin deficiency (CD).

Building on the success of our previous meetings, this symposium will reunite our clinical and research consortium to share updates on the substantial progress being made, welcome new members embarking on fresh research projects, and allow us to hear new approaches from researchers who are not yet part of our consortium.

Beyond our focus on citrin deficiency, this symposium will also explore broader issues in the rare disease space, as our Foundation is increasingly becoming a driving force in bringing together diverse stakeholders to tackle the complex challenges of developing treatments for rare disease patients.

Through strategic research funding and partnerships with scientists, clinicians, and biotechnology companies, we aim to drive innovative solutions for our patients. This symposium will therefore serve as a platform to showcase our scientific and clinical advancements, to further facilitate knowledge exchange, stimulate discussions, and inspire new ideas.

The scientific program will span two and a half days, featuring cutting-edge research and clinical developments across various sessions, including:

• Biochemical mechanisms and pathogenesis
• New preclinical models
• Novel diagnostic and bio-profiling methodologies
• Advances in therapies: gene therapy, mRNA therapy, and small molecules
• Clinical trial readiness
• Natural history studies, heterogeneity, genetics, and environmental factors
• Industry developments and perspectives

In addition to presentations and poster sessions, curated social activities will provide attendees with opportunities to connect and network.

This year’s symposium will take place at the Møller Institute, within the University of Cambridge—a globally renowned institution at the forefront of scientific discovery, offering an intellectually stimulating and scenic setting.

We remain uncompromising and resolute in our mission to develop a cure for CD. Your active participation in the symposium will be invaluable in advancing our vision and, in the process, contributing to progress in the broader rare disease space.

We look forward to welcoming you.

Barbara Yu and Yen How Tai
Co-Founders, Citrin Foundation