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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, we are also working with charities, industry and other universities to ensure that we are reaching the right people for our 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 Nucleic Acid Therapies for rare disease.

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