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 Sponsors
Symposium Programme
Wednesday, 23 September 2026
| Time | Event / Speaker |
| 13:30 – 14:00 | Registration and Coffee |
| 14:00 – 14:10 | Opening Remarks Prof Matthew Wood, Oxford-Harrington Rare Disease Centre |
| SESSION 1 | Translational Breakthroughs in Rare Disease Therapy Development Chair: Prof Stephan Sanders, University of Oxford |
| 14:10 – 14:40 | CRISPR Cures On-Demand at Nationwide Scale: a Tractable Goal for 2030 Prof Fyodor Urnov, UC Berkeley |
| 14:40 – 15:00 | Gene Therapy for Huntington's Disease Prof Sarah Tabrizi, University College London |
| 15:00 – 15:30 | Oligonucleotide Therapies for Rare Cardiac Muscle Diseases Prof Metin Avkiran, British Heart Foundation Prof Hugh Watkins, University of Oxford Dr Martin Murphy, Cora Biosciences |
| 15:30 – 16:00 | Break |
| SESSION 2 | Science, Policy and Funding Innovations for Rare Disease Therapy Development |
| 16:00 – 16:40 | Panel Discussion Baroness Blackwood, Health Data Research Service (Chair) Dr Harriet Holme, Weatherden Dr Kath Bainbridge, Department of Health and Social Care Julian Beach, Medicines and Healthcare products Regulatory Agency Dr Melanie Ivarsson, Health Data Research Service Claire Pimm, LifeArc |
| 16:40 – 17:00 | How is GSK Approaching AI and Data-based R&D Including in Rare and Complex Disease? Dr Tony Wood, GSK |
| 17:00 – 17:20 | From Diagnostic Odyssey to New Treatments: a Moral Imperative Prof Patrick Chinnery, Medical Research Council |
| 17:20 – 17:45 | In Conversation Lord Cameron and Sir John Bell, Oxford-Harrington Rare Disease Centre Advisory Council |
| 17:45 – 19:00 | Networking Reception sponsored by Oxford Science Enterprises |
| 19:00 – 21:00 | Symposium Dinner in Trinity College Hall |
Thursday, 24 September 2026
| Time | Event / Speaker |
| 08:30 – 09:00 | Registration and Coffee |
| SESSION 3 | Therapy Development for Rare Neurological Diseases Chair: Prof Carlo Rinaldi, University of Oxford |
| 09:00 – 09:10 | Our Race to Find a Treatment for Our Children’s Ultra-rare Genetic Condition, DHDDS Mel Dixon, Cure DHDDS |
| 09:10 – 09:25 | From Mutation to Medicine – Developing ASO Therapy for SPTLC1-related Hereditary Sensory Neuropathy Prof Haiyan Zhou, University College London |
| 09:25 – 09:40 | Therapeutic Targets for Behavioural Problems: Insights from Autism Genetics Prof Matthew Anderson, Harrington Discovery Institute |
| 09:40 – 09:55 | ReNU Syndrome: From Gene Discovery to Therapy Development in a Newly Identified Neurodevelopmental Disorder Dr Alyssa Hill, University of Oxford and Dr Dora Markati, Roche |
| 09:55 – 10:10 | The Development of Nomlabofusp for the Treatment of Friedreich’s Ataxia Dr Rusty Clayton, Larimar Therapeutics |
| 10:10 – 10:25 | N-of-1 Therapeutics and the Future of Individualised Medicines Dr Tim Yu, Boston Children's Hospital |
| 10:25 – 10:55 | Break |
| SESSION 4 | Therapy Development for Rare and Childhood Cancers Chairs: Dr Julie Woda, Harrington Discovery Institute and Prof Isidro Cortes Ciriano, University of Oxford |
| 10:55 – 11:05 | Science, Emotion & Grit – Common Ground in Rare Disease Jonathan Bracey, MVA Society |
| 11:05 – 11:20 | Engineering Next-Generation T Cell Therapies for Cancer Prof Charles Gersbach, Duke University |
| 11:20 – 11:35 | Developing In Vivo CAR T Therapy for Treating Acute Myeloid Leukemia Prof Xianxin Hua, University of Pennsylvania |
| 11:35 – 11:50 | Development of Drug Switchable Chimeric Antigen Receptors for Childhood Solid Cancers Prof John Anderson, University College London |
| 11:50 – 12:05 | Borrowed Immunity: Turning COVID-19 Immune Memory into a Weapon against Rare Cancers Prof John Letterio, Angie Fowler Adolescent and Young Adult Cancer Institute, University Hospitals |
| 12:05 – 12:20 | C-Further: Pioneering Child-First Cancer Medicines Dr 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 | Panel Discussion Dr Kylie Bromley, Biogen Deborah Richards, Alexion AstraZeneca Nico Reynders, UCB Dr Nick Crabb, National Institute for Health and Care Excellence |
| SESSION 6 | Translating Academic Breakthroughs to the Clinic Chair: Dr Claire Brown, Oxford Science Enterprises |
| 14:15 – 14:30 | Linking Preclinical Discovery to First in Man Studies: The Example of Duchenne Muscular Dystrophy Prof Francesco Muntoni, University College London |
| 14:30 – 14:45 | Transforming Rare Respiratory Disease Outcomes Using Inhaled Gene Therapy David Hipkiss, AlveoGene |
| 14:45 – 15:00 | The Commercialisation Predicament for Rare Disease Drug Development - Can We Design a New Paradigm? Prof Moin Saleem, University of Bristol |
| 15:00 – 15:15 | Targeting RNA Regulatory Elements to Increase Protein Expression |
| 15:15 – 15:45 | Break |
| SESSION 7 | Global Delivery and Equitable Access to N-of-1 Therapies Chair: Marlen Lauffer, University of Oxford |
| 15:45 – 15:57 | The LifeArc Rare Therapies Launch Pad – Driving the Switch from Research to Treatment Dr Dan O’Connor, Association of the British Pharmaceutical Industry |
| 15:57 – 16:09 | From Gene to Patient - What it Takes to Build a Therapy for One Patient Dr Roxana Redis, IQVIA Laboratories |
| 16:09 – 16:21 | WinQure Medicine – Antisense Oligonucleotide Therapies for Patients with Ultra-rare Diseases Dr Stephanie Manton, WinQure |
| 16:21 – 16:33 | From Bespoke Therapies to Sustainable Systems: Implementing N=1+ Genetic Medicines in a Public Hospital Dr Gregory Costain, The Hospital for Sick Children, Toronto |
| 16:33 – 16:45 | Role of the Hospital Pharmacy in N=1 Oligo Therapy Dr Martin Pool, Leiden University Medical Center |
| 16:45 – 17:15 | Panel Discussion |
| 17:15 – 17:20 | Closing Remarks Prof Matthew Wood, Oxford-Harrington Rare Disease Centre |
| 17:20 – 18:15 | Networking Reception |
| END |