Scientific Advisory Board

Sir Gordon Duff, M.D., Ph.D., is the President of the United in Diversity Foundation working towards the UN’s Sustainable Development Goals and creating an international campus at Kura Kura Island, Bali. He is co-chair (with Prof Andrew Lo, MIT) of the recently launched $30 billion ‘Health of Nations’ investment fund. He previously served as the Principal of St. Hilda’s College at the University Oxford and as Chair of the UK’s Committee on Safety of Medicines and its Biologicals and Vaccines Sub-committee as well as inaugural Chair of the UK’s Commission on Human Medicines. Sir Gordon is an expert on healthcare and UK science policy with a special interest in the genetics of the inflammatory response and was co-founding editor of the international research journal CYTOKINE, advisory editor to the Human Genome Organisation (HUGO) journal, and is Past-President of the International Cytokine Society. He was Knighted for services to Public Health in 2007, has more than 450 peer-reviewed publications, and was listed in the ‘Top 100’ working scientists in the UK Science Council’s list for 2014. He was also previously Chair of the Biotechnology and Biological Science Research Council (BBSRC) and of the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), and so brings deep insights and a regulatory perspective of biopharma, drug discovery and drug development.

Annemieke is Professor of Translational Genetics at Leiden University as well as President of the Oligonucleotide Therapeutics Society (OTS) and chair of TREAT-NMD; a network for the neuromuscular field that provides an infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Annemieke is a world leading expert in exon skipping therapies and played an important role in the development of the antisense mediated exon skipping therapy for Duchenne muscular dystrophy already during her PhD research, and remains a scientific advisor for several Duchenne organizations. Her work to date has resulted in a deep knowledge about potential pitfalls in oligonucleotide therapeutic drug discovery and development, and as President of OTS Annemieke is well respected in the whole oligonucleotide therapeutics field.

Tom Brown is Professor of Nucleic Acid Chemistry in the Departments of Chemistry and Oncology at Oxford University. His research interests centre on oligonucleotide synthesis and applications of oligonucleotides in biology and medicine (diagnostics and therapeutics). His group has recently worked on the synthesis of oligonucleotides containing artificial backbones (e.g. triazoles) and on chemically modified CRISPR RNA. He is co-inventor of several technologies for genetic analysis and co-founder of three Biotech companies including ATDBio (synthesis of modified oligonucleotides). He has published around 450 research papers and patents. Awards including the Royal Society of Chemistry (RSC) Josef Loschmidt prize, the RSC award for Nucleic Acid Chemistry, the RSC prize for Interdisciplinary Research, Chemistry World entrepreneur of the year (2014) and BBSRC Innovator of the Year (2016). He was also presented with a lifetime award for external engagement and promoting impact by Oxford University. Tom is a Fellow of the Royal Society of Edinburgh and a Fellow of the Royal Society of Chemistry. He is former President of the Chemistry Biology Interface Division of the RSC, Editor-in-Chief of the RSC Book series on Chemical Biology and was co-Chair of the 2018 EuCheMS congress in Liverpool, UK. He recently completed a four-year stint as Associate Head of the Chemistry Department at Oxford University with responsibility for Research.

Henry is Professor of Medicine at Columbia University and has been so for the last 35 years, and a past Director for the Irving Institute for Clinical & Translational Research. Henry is an active senior investigator of cardiovascular disease and lipoprotein metabolism research with detailed knowledge about Lp(a) (the target for our pipeline project SLN360) but also all the drug development in the cardiovascular disease and lipoprotein metabolism field that has preceded it – the successes and the failures in a complicated area of disease. Henry has over 300 peer reviewed and invited publications and serves as the Chief Medical Officer for the Lp(a) Foundation, a key partner in development of drugs targeting Lp(a).

John is professor of Haematology and Consultant Haematologist at the University College London Hospitals in London, UK and head of the joint Red Cell Unit for UCLH and Whittington Hospitals. He has served as scientific adviser to the British Society of Haematology, the UK Thalassaemia Society, the Thalassaemia International Federation (TIF), and to grant review and advisory panels at the NIH in Bethesda, MD, USA. He has published more than 350 peer-reviewed articles and made numerous contributions to books, as well as clinical guidelines and other medical articles. John’s clinical and research focus has been treatments of thalassaemia and sickle cell disorders, with particular reference to iron overload, the mechanisms of iron chelation, the speciation and uptake of non-transferrin-bound iron (NTBI) species, the molecular basis of iron homeostasis in health and disease, and the actions and toxicities of mixed-ligand chelation therapies. As a world leading hematology KOL John brings essential practical knowledge relevant for our pipeline program SLN124.

Jenny Sims, Ph.D., has a degree in Biochemistry and a PhD in Pharmacokinetics and Toxicology. She has more than 30 years of experience in non-clinical drug development, with a focus on biotechnology products (monoclonal antibodies, antibody drug conjugates, protein drug conjugates, therapeutic proteins, novel scaffolds, DNA vaccines and cell & gene therapies). 

Jenny worked for 12 years as an Expert Preclinical Assessor in the Clinical Trials Unit and Biotechnology Group at the MHRA and was a member of many CHMP Rapporteur/Co-Rapporteur assessment teams for biopharmaceuticals and scientific advice procedures. She then became Head of Project Safety Assessment, Preclinical Safety at Novartis Pharma AG. Jenny then Headed up the BioPharma Safety Assessment Group at MedImmune before returning to Novartis to take on the role of Head of Translational Sciences and Safety, in the Novartis Biologics group.

In 2012, Jenny established Integrated Biologix GmbH, to provide independent non-clinical development consultancy for biopharmaceuticals and advanced therapy products, working with early start-ups and small/large companies at all stages of the drug-development cycle. Jenny has been involved in many successful clinical trial and BLA/CTD approvals and has experience of supporting projects at Health Authority Scientific Advice meetings, FDA advisory committees and CHMP oral hearings. She is a regular member of company Scientific Advisory Board meetings.

Jenny was the MHRA/UK delegate to the CHMP Safety Working Party and MHRA observer on the UK Gene Therapy Advisory Committee (GTAC). She was a member of the Expert Working Groups for ICHS6/ICHS6R(1), ICHS8, ICHM4(S) and was part of the UK industry review team post-Tegenero which recommended greater consideration of the pharmacological active dose (PAD) and MABEL approach for First in Human trials.

John Whittaker, Ph.D., is currently Director of the MRC (Medical Research Council) Biostatistics Unit (BSU). He previously served as VP in Human Genetics at GlaxoSmithKline (GSK) where he led GSK’s work to identify the causal genes and mechanisms underlying genetic association signals. He also led for GSK several public-private partnerships related to the HG (Human Genetics) remit of identifying high quality drug targets founded on genomic information, notably work with UK Biobank and Genomics England. Prior to this, he led both statistics and genetics groups at GSK.

Prior to his move to GSK in 2009, he held academic positions in several UK universities, most recently as Professor of Genetic Epidemiology and Statistics at the London School of Hygiene and Tropical Medicine, where he led a group working on statistical problems in biomedicine, particularly statistical genetics/genetic epidemiology. He currently holds honorary chairs at LSHTM and at the Institute for Medical Informatics at UCL.