The Challenge
Nucleic Acid Therapies (NATs) are genetic medicines that treat the causes of disease and have the potential to transform healthcare. They will provide personalized treatments for a vast range of areas of unmet need in human health.
The NAT Delivery Challenge consortium (TransNAT) aims to develop novel methodologies and platforms to more effectively target oligonucleotides to the specific parts of the body where they are needed. These therapies have the potential to treat any rare or common diseases with a genetic cause.
Neurological, neuromuscular and cardiovascular diseases in particular devastate lives and create a very significant economic and social burden across the entire global population. NATs have become a reality over the last decade with multiple medicines being approved for use. However, many challenges remain particularly for diseases outside the liver and for tissues or organs not easily accessible to local drug delivery. The challenge of NAT delivery put simply is to deliver the drug effectively across the cell membrane into the appropriate part of the cell at sufficient levels for activity and without causing safety concerns.
The Nucleic Acid Therapy Accelerator (NATA) has awarded £8m to a consortium led by Professor Matthew Wood at the University of Oxford to develop new methods for delivering these oligonucleotide therapies. The NATA Hub is a unit funded by UK Research & Innovation (UKRI), via Medical Research Council as a UK government strategic priority. Its goal is to advance the development of nucleic acid therapies (NATs) by working with world-leading academics, clinicians, companies and charities. It has funded two Research Challenges: one on NAT delivery and another on manufacturing. The Delivery Challenge has a funding contribution from LifeArc.
“We are thrilled to be working with the consortium led by the University of Oxford, that brings together world renowned experts in the field of nucleic acid therapy. We have now funded two consortia that, over the next 3-4 years, will significantly advance the field of oligonucleotide synthesis and delivery, this represents an important milestone for the UK scientific community and NATA”
Professor Nick Lench, NATA Executive Director
“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.
Sir John Bell, Oxford’s Regius Professor of Medicine