The Medical Research Council (MRC) and National Institute for Health and Care Research (NIHR) have committed ca. £14m to establish a UK Rare Diseases Research Platform, comprising 11 "nodes" based in universities across the UK. Each node is working on a research project that is either focused on one disease area or across multiple rare diseases, and whilst the nodes are based at universities, they are also working with charities, industry and other universities to ensure that they are reaching the right people for the research. We are the UPNAT Node ("Establishing a UK Platform of Nucleic Acid Therapy for Rare Disease").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 NATs for RD. We are focused on:1. Publishing guidelines for NAT design and pre-clinical development2. Enhancing the UK's capabilities in NAT scale-up synthesis & toxicology studies for Rare Disease3. Streamlining NAT clinical trial process and regulatory approval
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