The UPNAT Inaugural Symposium will take place at the UCL GOS Institute of Child Health (Kennedy Lecture Theatre), 30 Guildford Street, London, WC1N 1EH on 27 June 2024 from 10am to 5pm.
The aim of the UPNAT Node is to bring together relevant stakeholders to establish a national network promoting knowledge-exchanging and innovation in pre-clinical development and rapid clinical translation of Nucleic Acid Therapies for rare disease.
The agenda will include sessions on:
- Real-world experience from patients & clinicians
- Piotr Kosla (Parent, PACS2 Research Foundation, and Simons Searchlight Community Advisory Committee member)
- Francesco Muntoni (Chair of Paediatric Neurology, UCL)
- Target selection
- Stephan Sanders (Professor of Neurogenetics, Oxford)
- Ana Lisa Taylor Tavares (Clinical Lead for rare disease research, Genomics England)
- Clinical trials and regulatory path
- Sarah Tabrizi (Professor of Clinical Neurology, UCL)
- Daniel O’Connor (Director of Regulatory and Early Access Policy, Association of the British Pharmaceutical Industry)
- Willeke van Roon-Mom (Professor of Human Genetics, Leiden University)
- Panel discussion chaired by Paul Gissen (Clinical Professor of Paediatric Metabolic Medicine, UCL Great Ormond Street Institute of Child Health)
