UK Platform of Nucleic Acid Therapy for rare disease treatment


Lead Contact

Dr Haiyan Zhou

University College London


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

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. 


The UPNAT node will:
  • Bring together scientists, geneticists and clinicians to generate experimental guidelines and promote knowledge exchange on NAT development for RD, and promote communications, public engagement and raise awareness for experimental NAT development among RD patients, families, charities, advocacy groups and clinical centres.
  • Link and seek further collaboration with academic groups, industry and international consortia to explore NAT treatments in diverse disease areas, with particular emphasis on rare disease.
  • Work closely with regulatory authorities to establish and publish clear guidance on RD NAT preclinical development, clinical trial design principles and regulatory approval applications, to streamline the process and accelerate the clinical translation of such therapies in an efficient and safe manner.


Who We Work With