UPNAT Node Annual Symposium 2025

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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The agenda will include sessions on:

Target Selection Guidelines

• Dr Ella Whittle (UPNAT Node / UCL) will present progress on the UK Target Selection Guidelines
• Dr Amy McTague (GOSH) will provide a case-study of the assessment process using the Gene-STEPS study
• Dr Marlen Lauffer (Dutch Center for RNA Therapeutics) will set in focus the alignment of therapeutic eligibility assessments globally
• Dr Alban Ziegler (University Hospital of Toulouse) will discuss personalised oligonucleotide antisense for nano-rare diseases and the role for medical geneticists

Accelerating clinical translation of Nucleic Acid Therapies

• Barney Hill (University of Oxford) will present the OligoAI machine learning tool to enhance accurate modelling of ASOs to accelerate preclinical development
• Rachel Smith (Parexel) will share her experience working in every development phase of rare disease, RNA, cell and gene therapy with biotech and large pharma
• Jeff Milton (La Jolla Labs) brings his experience from industry to drive scientific innovation and platform development

Public and Patient Involvement and Engagement:

• Claire Andersen, PhD (UNIQUE, Understanding Genes & Chromosomes) will provide an update about their charity activity and collaboration with UPNAT

UPNAT Node Partners

• Assoc Prof Peter Oliver (Nucleic Acid Therapy Accelerator - NATA) will provide and update on NATA translational pipelines and recent progress on ASO / siRNA therapies for neurological disorders
• Dr Andrew Roddam (EveryONE Medicines - EOM) will introduce EoM’s programme and explore a scalable and sustainable solution for individualized therapies
• Claire MacLeod (Centre for Process Innovation - CPI) will review strategies for Good Manufacturing Practice (GMP) manufacturing of oligonucleotide therapeutics

UK Rare Disease Framework

• Prof Matt Brown (Genomics England) will deliver a keynote presentation on Translational Genomics and Rare Diseases at Genomics England
• Dr Kath Bainbridge (Department of Health and Social Care), who helped shape the 2025 Action Plan, will review the rare disease landscape within DHSC and NHS England

+Panel discussions with Symposium speakers and invited guests

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Host or Publisher UK Platform of Nucleic Acid Therapies - UPNAT