Event
Oxford-Harrington Rare Disease Centre Symposium 2026
23rd - 24th September, 2026
- Additional Details:
The Oxford-Harrington Rare Disease Centre Symposium will take place in Trinity College, Oxford, in September 2026. The OHC Symposium is at maximum capacity and registration is now closed.
Symposium Programme
Wednesday, 23rd of September, 2026
| Indicative Time | Event / Speaker |
| 13:30 – 14:00 | Registration and Coffee |
| 14:00 – 14:10 | Opening Remarks - Matthew Wood, Director, Oxford-Harrington Rare Disease Centre |
| SESSION 1 | Translational Breakthroughs in Rare Disease Therapy Development |
| 14:10 – 14:40 | Gene Editing for Rare Metabolic Diseases - Fyodor Urnov, UC Berkeley - Rebecca Ahrens-Nicklas, Children's Hospital of Philadelphia |
| 14:40 – 15:00 | Gene Therapy for Rare Neurological Diseases - Sarah Tabrizi, University College London |
| 15:00 – 15:30 | Oligonucleotide Therapies for Rare Cardiac Muscle Diseases - Metin Avkiran, British Heart Foundation - Hugh Watkins, University of Oxford - Martin Murphy, Cora Biosciences |
| 15:30 – 16:00 | Break |
| SESSION 2 | Science, Policy and Funding Innovations for Rare Disease Therapy Development |
| 16:00 – 16:50 | Policy Expert Panel Discussion - Baroness Blackwood, Health Data Research Service (Chair) - Harriet Holme, PCD Research; Weatherden - Kath Bainbridge, Department of Health and Social Care - Julian Beach, Medicines and Healthcare products Regulatory Agency - Melanie Ivarsson, Health Data Research Service - George Orphanides, LifeArc |
| 16:50 – 17:10 | GSK and How Rare Disease Can Be a Mechanistic Model for Patient Selection in Common Diseases - Tony Wood, GSK |
| 17:10 – 17:30 | Advancing Rare Disease Science and Medical Research Council Strategy - Patrick Chinnery, Medical Research Council |
| 17:30 – 17:45 | Closing Remarks - Lord Cameron, Chair, Oxford-Harrington Rare Disease Centre Advisory Council |
| 17:45 – 19:00 | Networking Reception |
| 19:00 – 21:00 | Symposium Dinner in Trinity College Hall |
Thursday, 24th of September, 2026
| Indicative Time | Event / Speaker |
| 08:30 – 09:00 | Registration and Coffee |
| SESSION 3 | Therapy Development for Rare Neurological Diseases |
| 09:00 – 09:10 | Mel Dixon, Cure DHDDS |
| 09:10 – 09:25 | Haiyan Zhou, University College London |
| 09:25 – 09:40 | Matthew Anderson, Harrington Discovery Institute, University Hospitals |
| 09:40 – 09:55 | Alyssa Hill and Dora Markati, University of Oxford |
| 09:55 – 10:25 | Tim Yu, Boston Children's Hospital |
| 10:25 – 10:55 | Break |
| SESSION 4 | Therapy Development for Rare and Childhood Cancers |
| 10:55 – 11:05 | Jonathan Bracey, MVA Society |
| 11:05 – 11:20 | Charles Gersbach, Duke University |
| 11:20 – 11:35 | Xianxin Hua, University of Pennsylvania |
| 11:35 – 11:50 | John Anderson, University College London |
| 11:50 – 12:05 | John Letterio, Angie Fowler Adolescent and Young Adult Cancer Institute, University Hospitals |
| 12:05 – 12:20 | Lone Friis, LifeArc |
| 12:20 – 13:35 | Lunch |
| SESSION 5 | Biopharma Industry Perspectives on Translation, Policy and Patient Access to Rare Disease Medicines |
| 13:35 – 14:15 | - Clive Cookson, The Financial Times (Chair) - Kylie Bromley, Biogen - Deborah Richards, Alexion AstraZeneca - Nico Reynders, UCB - Nick Crabb, National Institute for Health and Care Excellence |
| SESSION 6 | Translating Academic Breakthroughs to the Clinic |
| 14:15 – 14:30 | Francesco Muntoni, University College London |
| 14:30 – 14:45 | David Hipkiss, AlveoGene |
| 14:45 – 15:00 | Moin Saleem, University of Bristol |
| 15:00 – 15:15 | Stuart Milstein, Orfonyx Bio |
| 15:15 – 15:45 | Break |
| SESSION 7 | Global Delivery and Equitable Access to N-of-1 Therapies |
| 15:45 – 17:15 | - Marlen Lauffer, University of Oxford (Chair) - Dan O’Connor, Association of the British Pharmaceutical Industry - Roxana Redis, IQVIA Laboratories - Martin Pool, Leiden University Medical Center - Gregory Costain, The Hospital for Sick Children, Toronto, Canada - Stephanie Manton, WinQure |
| 17:15 – 18:15 | Networking Reception |
| * The indicated times are subject to change. |