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 in addition to vaccination. NATs are expected to transform personalised medicine because they can be tailored to the DNA sequences of individual patients.
Although multiple NAT medicines have been approved for use in Europe and the US, challenges remain in safely delivering the drug to the organ or tissue where it is needed, especially the brain, heart and muscle. The NATA Delivery Research Challenge was launched to overcome delivery obstacles to fulfilling the potential of this emerging class of drugs. Responding to the announcement,
Professor 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.'
Read the full story on the Department of Paediatrics website.