OHC symposium focuses on rare disease research, therapy
Academic, biotech, pharma leaders discuss new treatment strategies
The Oxford-Harrington Rare Disease Centre (OHC) is hosting its biannual symposium on rare disease therapy development drivers, bringing together experts from academia, biotechnology, pharmaceuticals, venture capital, and patient advocacy to discuss the latest research, therapeutic strategies, and collaboration efforts for rare diseases such as aromatic L-amino acid decarboxylase (AADC) deficiency.
The event, held Sept. 24-26 at the University of Oxford’s Trinity College, ”is a unique opportunity to bring the international rare disease community together to recognise scientific progress and innovation and foster collaboration among leaders in the field,” OHC Director Matthew Wood, MD, PhD, professor of neuroscience at the University of Oxford, said in an OHC press release. OHC is a partnership between the University of Oxford and Harrington Discovery Institute at University Hospitals in Cleveland.
Daily sessions at the three-day event explore topics including therapeutic genomics, drug development, and early intervention for neuromuscular diseases. Speakers include Baroness Nicola Blackwood, chair of Genomics England; Jonathan Stamler, MD, president and co-founder of the Harrington Discovery Institute; and Julia Vitarello, CEO of Mila’s Miracle Foundation. Fyodor Urnov, PhD, professor and scientific director at the University of California Berkeley’s department of molecular and cell biology, speaks on advancements in gene-editing therapies for rare diseases.
Commitment to rare disease research
In addition to therapeutic genomics, an area of medicine focused on leveraging knowledge of genes and their variations to develop targeted therapies, the event features discussions on the Rare Therapies Launch Pad, a pilot program designed to develop a pathway for children with rare conditions to access therapies made specifically for them, as well as talks on partnerships on rare disease drug development, early intervention for neuromuscular diseases, and therapies for rare and childhood cancers.
The program includes an update on OHC’s Therapeutics Accelerator, a partnership backed by up to 200 million pounds (about $267 million) in funding.
More than 400 million people around the world are living with rare diseases, though only 5% of those diseases have treatments approved by the U.S. Food and Drug Administration (FDA) or European Medicines Agency, according to OHC.
The symposium brings 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,” said Wood.
About the Author
