UK Platform of Nucleic Acid Therapy for rare disease treatment

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.