Professor Francesco Muntoni, University College London
Professor Nick Lench, MRC Harwell
Dr Alice Davidson, University College London
Professor Mina Ryten, UK Dementia Research Institute, Cambridge University
Dr Carlo Rinaldi, University of Oxford
Dr Ella Francesca Whittle, University College London
Catherine Ryan (Project Manager), University College London
Nucleic acid therapeutics (NATs) offer great potential to treat rare diseases (RDs) by targeting the specific genetic cause. The huge increase in NAT clinical trials and approved therapies in the last few years clearly demonstrates their role in research to transform new ideas into new treatments and unique opportunities for preclinical and clinical studies, in which the UK has a high level of expertise.
UPNAT Node aims to further promote the development of NATs for patients with Rare Disease in the UK, by creating a national network including scientists, clinicians, geneticists, patient advocates and charities, industrial partners, international non-profit organizations and regulatory bodies, to facilitate the development of NAT for RDs.
UPNAT intends to address a number of challenges, including 1) a lack of a national networks for knowledge and expertise sharing between centres leading NAT preclinical and clinical development 2) a clear path for systematically linking patients who have unique mutations to NAT expertise and 3) the need for continuous dialogue between regulators and researchers to streamline the process of regulatory approvals and accelerating the clinical translation of RD-specific NATs.
Dr Haiyan Zhou is a Professor of Genetic Medicine at University College London (UCL) and the UK Harrington Rare Disease Scholar. Her research focuses on the preclinical development and clinical translation of nucleic acid therapy (NAT) for rare genetic disorders. Prof. Zhou serves as the deputy theme lead of the novel therapy theme in the NIHR Biomedical Research Centre at UCL Great Ormond Street Hospital (GOSH BRC) and leads the individualized RNA therapy program for paediatric rare diseases. Prof. Zhou is the Director of the UK Platform of Nucleic Acid Therapy (UPNAT Node) for rare disease, a specialist rare disease node supported by the UK Medical Research Council (MRC) and NIHR. Prof. Zhou is the principal investigator in a series of research projects on the development of RNA therapeutics for a wide range of genetic diseases. She is also the director of the MSc Personalized Medicine and Novel Therapies Programme at UCL.
Prof. Haiyan Zhou is a member of the World Muscle Society, the Oligonucleotide Therapeutic Society, and the American Society of Gene and Cell Therapy. She is also the UK representative and management committee member of the European Cooperation in Science and Technology (e-COST) on ‘Delivery of Antisense RNA Therapeutics’.
Prof. Zhou holds a MD-PhD degree in clinical medicine and skin pharmacology from Peking Union Medical College.
