Accelerating the formation of international consortia to tackle some of the most pressing challenges to nucleic acid therapy development
The Research Challenge fund will enable and accelerate early stage innovation for nucleic acid therapy development, developing and disseminating platform technologies that will clear the way for the next generation of precision medicines.
By supporting international consortia of academic and industrial experts we will address barriers to the manufacture and delivery of synthetic nucleic acid medicines, opening new opportunities to treat more patients globally.
The £12M scheme is funded by the UK Strategic Priorities Fund and delivered by the Medical Research Council, part of UK Research and Innovation.
The Manufacture Research Challenge scheme is now open to expressions of interest and will close at 4pm on 10th June 2021. Further information is available on the UKRI Funding Finder.
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The promise of nucleic acid medicines has been showcased by recent high-profile regulatory approvals however for these medicines to truly instigate a paradigm shift in healthcare, major scientific and industrial challenges need to be addressed.
Research Challenge funding will support two consortia of academic and industry experts; one consortium will address barriers to the manufacture of synthetic nucleic acid therapeutics and one consortium will address barriers to precision delivery. Up to £6m of UKRI funding will be available to each consortium plus leveraged funds.
Manufacture Research Challenge
Manufacture of nucleic acid therapeutics is currently complex, expensive and resource intensive, limiting large-scale production of these medicines. Despite improvements in product separation and reductions in stereoisomer by-products during chemical synthesis, purity remains a challenge. New developments in solution-phase synthesis offer exciting opportunities to scale up, improve efficiency and reduce organic solvent use. Improved platforms for conjugation of targeting ligands hold promise for precision delivery and improvements in analytical characterisation remain crucial to regulatory confidence. This call will provide funding for a consortium to tackle these limitations.
The Manufacture Research Challenge is now open for expressions of interest until 10th June 2021. For further information on the opportunity’s scope and application process, go to the UKRI Funding Finder.
Delivery Research Challenge
The scope of the Delivery Research Challenge is currently being finalised through close engagement with the research and innovation community. Further information will be made available shortly.
These opportunities will be open to academic, SME and large industrial organisations based in the UK or abroad. The lead organisation must be UK-based. Projects must be collaborative - at least two collaborators must take grant, including at least one partner commercially active in the NAT space in the UK.
Proposals must articulate a substantial, ambitious programme of work with clear potential to catalyse a step-change in UK NAT development, having downstream translatability and wide-ranging industrial utility.
Assembled consortia will be able to partner with the NATA Hub, a new research facility on the Harwell Science Campus, UK. NATA Hub will offer specialist NAT synthesis, characterisation and screening technology and expert advice to support the consortia.
Contact us at
The NATA’s executive team are working closely with our internationally-leading scientific advisory group members to shape our Research Challenges:
Paul Bolno (Chair)
President and CEO, WaVe Life Sciences (US)
Dr. Bolno has served as President and CEO of Wave Life Sciences since 2013 and oversaw the company’s initial public offering in 2015. Prior to joining Wave, he was Vice President, Worldwide Business Development—Head of Asia BD and Investments, as well as Head of Global Neuroscience BD, at GlaxoSmithKline (GSK). He also served as a Director of Glaxo Wellcome Manufacturing, Pte Ltd. in Singapore. Dr. Bolno joined GSK as Vice President, Business Development for the Oncology Business Unit, where he helped establish GSK’s global oncology business and served as a member of the Oncology Executive Team, Oncology Commercial Board and Cancer Research Executive Team. Prior to GSK, he served as Director of Research at Two River LLC, a healthcare private equity firm.
Chief Scientist of New Therapeutic Modalities and Head of Oligonucleotide Discovery, Astrazeneca (Sweden)
Dr. Shalini Andersson obtained a PhD in organic and analytical chemistry in 1989 from Linköping University. After a postdoc at the Institute for Pharmaceutical Chemistry in Münster, Germany, Shalini joined Linköping University as a Senior lecturer in Organic Analytical Chemistry and became an Associate Professor (Docent) in 1996. Shalini joined AstraZeneca in 1997 as a senior researcher, subsequently holding numerous management roles including Senior Director Drug Metabolism and Pharmacokinetics, Head of Enabling Technologies and Head of Lead Optimization, Medicinal Chemistry. Shalini is currently Chief Scientist New Therapeutic Modalities and Head of Oligonucleotide Discovery, in AstraZeneca. Shalini is also a member of the joint steering committees for the collaborations with Moderna Therapeutics, Ionis Pharmaceuticals, MiNA Therapeutics and Silence Therapeutics on discovery and development of nucleic acid therapeutics. Shalini is author of >50 papers and patents and serves on the board of OligoNucleotide Therapeutics Society and EU COST action on Delivery of Antisense RNA Therapeutics.
Professor, Department of Biochemistry, UT Northwestern (US)
Dr. David Corey received his B.A. degree in chemistry from Harvard University and his Ph.D. degree in chemistry from the University of California Berkeley, working under the supervision of Dr. Peter Schultz to develop sequence specific engineering nucleases. Dr. Corey joined the UT Southwestern Pharmacology Department in 1992. He was promoted to Associate Professor with tenure in 1998 and Full Professor in 2003. In 2014, he was named the Rusty Kelley Professor of Medical Sciences. He is also a member of the Department of Biochemistry and the Simmons Cancer Center. Dr. Corey is an Executive Editor for Nucleic Acids Research and is on the Editorial Board of Cancer Research, Molecular Therapy Nucleic Acids, and Oligonucleotide Therapeutics. He is the author of more than 147 papers and has received funding from the Welch Foundation, NIH, the McKnight Award for Neuroscience Research, the American Heart Association, the Cure Huntington Disease Initiative, and the Freidreich's Ataxia Association.
Senior Vice President of Product Development and Supply, GSK (UK)
Dr. Annette Doherty has 32 years of international experience working within the pharmaceutical sector, leading Research and Development groups worldwide, including at Pfizer and GlaxoSmithKline (GSK). She is currently Senior Vice President, Global Head of Product Development and Clinical Supply at GSK. She has published more than 100 scientific manuscripts and written 19 reviews in the research areas in which she has worked. She is co-inventor of over 30 patents. In 2007 she received an honorary degree of Doctorate of Science from the University of Greenwich for her scientific leadership in research and contributions to education and industry/academic partnerships. She has served as a member of the Cambridge University Chemistry Strategic Advisory Panel from 2013-2019 and was a Member of the Cambridge Biomedical Research Centre Scientific Advisory Board in 2019.
Muthiah (Mano) Manoharan
Senior Vice President of Drug Innovation Chemistry, Alnylam (US)
Dr. Muthiah (Mano) Manoharan serves as a Senior Vice President and a Distinguished Research Scientist at Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA. Dr. Manoharan joined Alnylam in 2003. He built the chemistry group at Alnylam and pioneered the discovery and development of RNA interference-based human therapeutics. Dr. Manoharan has had a distinguished career as a world-leading chemist in the areas of oligonucleotide chemical modifications, conjugation chemistry, and delivery platforms (lipid nanoparticles, polymer conjugates, and complex-forming strategies). He is an author of more than 200 publications (nearly 37,000 citations with an h-index of 85 and an i10-index of 329) and over 400 abstracts, as well as the inventor of over 225 issued U.S. patents.
Chief Scientific Officer, Intellia Therapeutics (US)
Dr. Laura Sepp-Lorenzino oversees all platform and pipeline research activities across in vivo and ex vivo (engineered cell therapy) areas as Intellia’s Chief Scientific Officer. Before joining Intellia, she was vice president, Head of Nucleic Acid Therapies, Research, and member of the External Innovation team at Vertex Pharmaceuticals, Inc. She also served as vice president, entrepreneur-in-residence at Alnylam Pharmaceuticals, Inc., a leader in the development of RNAi Therapeutics. At Alnylam, she was responsible for the Hepatic Infectious Disease Strategic Therapeutic Area, championed extra hepatic siRNA delivery, and was active in licensing and partnering. She is a member of the board of directors of Taysha Gene Therapies, and serves on the scientific advisory boards of Thermo Fisher Scientific and Lodo Therapeutics.
Senior Advisor, Abingworth Investors (UK)
Prior to joining Abingworth he held positions at Cantab Pharmaceuticals as Senior Vice President, Research, the Glaxo Institute for Molecular Biology and the Institute of Child Health, UCL. In addition, John is on the boards of Adaptate Biotherapeutics and Microbiotica, and serves on numerous academic committees including the MRC Translational Research Group committee, the Biomedical Catalyst Major Awards Committee, Cancer Research UK CoLC Science Advisory Board, the Crick Institute Translation Advisory Group and the Sanger Centre Translational Committee. He has a PhD in Immunology from the University of Glasgow and has published more than 30 papers in peer-reviewed journals including Science.
Professor of Clinical Neurology, UCL Queen Square Institute of Neurology (UK)
Sarah Tabrizi is Director of the UCL Huntington’s Disease (HD) Centre, Joint Head of Department Neurodegenerative Disease at the UCL Queen Square Institute of Neurology, a Principal Investigator at the UK Dementia Research Institute, and Consultant Neurologist at the National Hospital for Neurology and Neurosurgery. In addition to a basic bench science programme focussing on basic cellular mechanisms of neurodegeneration in HD that can be harnessed for therapeutics, she also leads a large translational research programme that is working towards finding effective disease-modifying treatments for HD. She was global clinical PI on the world’s first gene targeting study for HD using anti-sense oligonucleotide therapy. Sarah was elected as a Fellow of the UK Academy of Medical Sciences in 2014. In 2017 she received the seventh Leslie Gehry Brenner Prize for Innovation in Science awarded by the Hereditary Disease Foundation. In 2018 she received the Cotzias Award from the Spanish Society of Neurology, and in 2019 the Yahr Award at the World Congress for Neurology and the Alexander Morison Medal from the Royal College of Physicians of Edinburgh.
Professor of Toxicology and MRC Toxicology Unit Director, University of Cambridge (UK)
Prof. Anne Willis obtained a PhD in Biochemistry from the University of London while working in the Imperial Cancer Research Fund laboratories (now CRUK) on DNA repair with Dr Tomas Lindahl. She then moved to Cambridge to work with Professor Richard Perham in the Department of Biochemistry, where she also held a Junior Research Fellowship and then a College Lectureship at Churchill College Cambridge. Anne was appointed to her first independent position as a Lecturer in the Biochemistry Department at the University of Leicester, progressing to Reader in 2002 and Professor in 2004, from 2000-2005 she held a BBSRC Advanced Fellowship. In 2004, she was appointed Director of Cancer Research Nottingham and Chair of Cancer Cell Biology, where she was based in the School of Pharmacy. From 2009-2013 Anne held a BBSRC Professorial Fellowship. In 2010 Anne became Director of the MRC Toxicology Unit. Anne was appointed as a member of the European Molecular Biology Organisation in 2015, and awarded an OBE for services to biomedical sciences and supporting the careers of women scientists.
Professor of Neuroscience | Deputy Head Medical Sciences Division, University of Oxford (UK)
Matthew Wood is a world-leading translational scientist. He is currently Professor of Neuroscience and Deputy Head of the Medical Sciences Division at the University of Oxford and directs the Laboratory of RNA biology and Neuromuscular Disease. He is the Director of two Oxford research centres – the MDUK Oxford Neuromuscular Centre and the Oxford Harrington Rare Disease Centre, reflecting his work to develop gene-based/oligonucleotide therapies and delivery technologies targeting neuromuscular and neurological diseases. He is the co-founder of three spin-out companies and also serves on the Board of the University of Oxford technology transfer company, Oxford University Innovation (OUI).
All advisory members provide independent advice to support the development of strategy and priority setting for NATA. The views of our members are provided in an independent capacity and do not necessarily represent the official views of their employers or host research organisations.