A consortium led by Matthew Wood, Professor of Neuroscience in the Department of Paediatrics and Director of the Oxford-Harrington Rare Disease Centre, has been awarded funds from UKRI via the Nucleic Acid Therapy Accelerator (NATA) based at Harwell.
Nucleic Acid Therapies (NATs) are a major new class of medicines that are expected to transform personalised medicine because they can be tailored to the DNA sequences of individual patients. Programmable nucleic acid-based therapeutics have come of age at pace during the pandemic, with billions of doses of mRNA COVID vaccines being administered globally. The emergence of these types of drugs, and their approval by regulatory agencies, offers significant potential to address unmet clinical need in many areas of human health.
Although multiple NAT medicines have been approved for use in Europe and the US, challenges remain in delivering the drug to the organ or tissue where it is needed, especially the brain, heart and muscle. Responding to the announcement, Prof. Matthew Wood said “This major award gives an outstanding international consortium the opportunity to focus on key scientific challenges in safely and specifically delivering nucleic acid therapeutics to diseased tissues. Our vision is to accelerate development of this field and bring about a transformation in the use of nucleic acid drugs to benefit patients affected by a wide range of rare and common diseases”.
The consortium includes an international group of academic experts in NAT chemistry, delivery and toxicology, and three key industry partners: AstraZeneca, Silence Therapeutics and Ionis Pharmaceuticals, which has five NAT drugs that have received regulatory approval globally. Oxford researchers include Tom Brown, Professor of Nucleic Acid Chemistry in the Department of Chemistry, and Matthew Wood’s group in the Institute of Developmental and Regenerative Medicine.
Sir John Bell, Oxford’s Regius Professor of Medicine, said of the award “This highly innovative programme aligns with the national priority to build capacity in nucleic acid therapeutics for human health, highlighted in the UK government’s Life Sciences Industrial Strategy and its substantial investment in the Nucleic Acid Therapy Accelerator. A number of nucleic acid drugs have been approved by regulators in recent years. The TransNAT consortium of internationally leading pharmaceutical and academic researchers, led by Oxford, will work to overcome the challenges of targeted drug delivery and drive growth in this important therapeutic area”.