Conferences & Events

NATA participates in UPNAT Inaugural Symposium at UCL Institute of Child Health

5 July 2024
A simulating panel discussion
Daniel O'Connor, Director of Regulatory and Early Access Policy from the Association of the British Pharmaceutical Industry, discusses the regulatory challenges associated with 'N of 1' therapeutics. From left to right: David Fischer, Chief Technology Officer, Discovery; Daniel O'Connor, Director of Regulatory and Early Access Policy, Association of the British Pharmaceutical Industry; Melanie Dixon, Trustee at Cure DHDDS.

NATA was pleased to participate in the UPNAT Inaugural Symposium, held last week at the UCL Institute of Child Health in London. This event marked a significant milestone in advancing nucleic acid therapies for rare diseases. 

The symposium was part of a larger initiative supported by the Medical Research Council and NIHR, which have committed approximately £14 million to create the Rare Diseases Research UK Platform. This platform includes 11 "nodes" across UK universities, with NATA being a key part of the UPNAT Node—dedicated to establishing a national network for nucleic acid therapies in rare diseases. 

The symposium featured insightful talks and two engaging panel discussions on "Nucleic Acid Therapy Target Selection in Rare Diseases" and "Clinical Trials and Regulatory Path." The active participation from the audience highlighted the importance of collaboration and innovation in this field. 

panel discussion ​
Panel discussion chaired by Jenny Taylor, Professor of Translational Genomics at the University of Oxford. From left to right: Stephen Sanders, Professor of Neurogenetics, Oxford; Willeke van Roon-Mom, Professor of Human Genetics, Leiden University; Ana Lisa Taylor Tavares, Clinical Lead for Rare Disease Research, Genomics England; Jenny Taylor, Professor of Translational Genomics, University of Oxford. 

NATA is excited to be part of this groundbreaking effort, working alongside UCL and the University of Oxford to develop a comprehensive roadmap for the translation and regulation of nucleic acid therapies. This partnership is a crucial step forward in our mission to improve treatment options for patients with rare diseases. 

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