The Oxford Harrington Rare Disease Centre (OHC) will host a biennial symposium on drivers of rare disease treatment development, bringing together experts from academia, biotechnology, pharmaceuticals, venture capital and patient advocacy to discuss the latest research, treatment strategies and collaborations for rare diseases such as aromatic L-amino acid decarboxylase (AADC) deficiency.
The event, held at Trinity College, Oxford from September 24-26, is “a unique opportunity to bring together the international rare disease community, recognize scientific advances and innovations, and foster collaboration among leaders in the field,” Matthew Wood, MD, professor of neuroscience at Oxford University and director of the OHC, said in a press release from the OHC. The OHC is a partnership between the University of Oxford and the Harrington Discovery Institute at University Hospitals Cleveland.
The three-day event will feature daily sessions covering topics such as therapeutic genomics, drug development and early intervention in neuromuscular diseases. Speakers include Baroness Nicola Blackwood, chair of Genomics England, Jonathan Stamler, MD, president and co-founder of Harrington Discovery Institute, and Giulia Vitarello, CEO of Mira’s Miracle Foundation. Dr Fyodor Urnov, professor and scientific director of the Department of Molecular and Cellular Biology at the University of California, Berkeley, will speak about advances in gene editing therapies for rare diseases.
Recommended reading
Rare Disease Research Initiative
The event will feature therapeutic genomics, a field of medicine focused on using knowledge about genes and their mutations to develop targeted therapies, as well as discussions on the Rare Therapies Launchpad, a pilot program designed to develop pathways for children with rare diseases to access therapies tailored to them; and talks on rare disease drug development, early intervention in neuromuscular diseases, and partnerships for therapies for rare and pediatric cancers.
The program also includes an update on OHC’s Therapeutics Accelerator, a partnership funded with up to £200 million (approximately $267 million).
According to the OHC, more than 400 million people worldwide live with a rare disease, but only 5% of them have treatments approved by the U.S. Food and Drug Administration (FDA) or the European Medicines Agency.
Wood said the symposium will bring together “global leaders from academia, industry, government and patient advocacy who share a commitment to translating science into tangible outcomes for rare disease patients and their families.”
