Event
Oxford-Harrington Rare Disease Centre Symposium 2024
24th - 26th September, 2024
The Oxford-Harrington Rare Disease Centre Symposium will take place on 24-26 September 2024 at Trinity College, Oxford. Please note, we have reached capacity for this event and registration is now closed.
For further information about accommodation and travel, please see the Cvent page.
The Symposium will explore how pioneering research, innovative treatment approaches, and collaborative efforts across biotech, pharma, academic institutions, funders, patient organisations, and regulators can enhance and accelerate the delivery of effective therapies for rare diseases.
The symposium programme offers updates on these activities and includes sessions covering a range of key topics in rare diseases including therapeutic genomics, drug development exemplars, early therapeutic intervention for neuromuscular diseases and paediatric cancer.
View the OHC Symposium 2024 Programme below.
- Venue:
- Trinity College, Oxford, UK
- Email:
- info@oxfordharrington.org
OHC Symposium 2024 Sponsors
Trinity College, Oxford
Tuesday 24th September | ||
|---|---|---|
| 15:30 | Registration and Tea | |
| 16:00 | Matthew Wood Director and Chief Scientific Officer, Oxford-Harrington Rare Disease Centre, University of Oxford Sir John Bell President, Ellison Institute of Technology | Opening Remarks |
| 16:15 | OPENING SESSION | Chair: Prof Matthew Wood, Oxford-Harrington Rare Disease Centre |
| 16:15 | Baroness Nicola Blackwood, Chair, Genomics England and Member of the House of Lords | Generation Genome: Integrating Rare Therapies into Healthcare |
| 16:30 | Julia Vitarello, Founder & CEO, Mila’s Miracle Foundation | From Mila to Millions |
| 16:45 | Prof Fyodor Urnov, Professor of Molecular & Cell Biology, University of California Berkeley | CRISPR Cures On-Demand at Nationwide Scale: An Actionable Path |
| 17:25 | Dr Jonathan Stamler, MD, President and co-Founder, Harrington Discovery Institute | Harrington Model and Accelerator |
| 17:40 - 18:40 | Drinks Reception | |
Wednesday 25th September | ||
|---|---|---|
| 8:30 | Registration and Coffee | |
| 8:55 | SESSION 1 Therapeutic Genomics | Chair: Prof Stephan Sanders, University of Oxford |
| 9:00 | Prof Stephan Sanders, Professor of Neurogenetics, University of Oxford | Therapeutic Genomics - Learning to Treat Rare Genetic Disorders at Scale |
| 9:20 | Dr Jasmina Kapetanovic, MRC Clinician Scientist, Consultant Ophthalmic Surgeon, University of Oxford | Genetic Therapies for Retinal Diseases |
| 9:40 | Prof James Davies, Associate Professor of Genomics, University of Oxford | Developing Therapeutic Genomics Approaches For Immunohaematology |
| 10:00 | Prof Sarah Tabrizi, Professor of Clinical Neurology & Neurogenetics, University College London | Developing Genetic Therapies for Huntington’s Disease - Challenges and New Developments |
| 10:20 | Prof Nicky Whiffin, Associate Professor and Sir Henry Dale Fellow, University of Oxford | De Novo Variants in the Non-coding snRNA RNU4-2 Cause a Frequent Neurodevelopmental Syndrome |
| 10:40 | Coffee Break (30 mins) | |
| 11:10 | SESSION 2 Rare Therapies Launch Pad | Chair: Dr Dan O'Connor, ABPI |
| 11:15 | Dr Dan O’Connor, Director, Regulatory Policy & Early Access, Association of the British Pharmaceutical Industry | The Rare Therapies Launch Pad: Opportunity to Innovate |
| 11:30 | Prof Timothy Yu, Professor of Pediatrics, Harvard Medical School | Scientific, Regulatory, and Ethical Implications of Precision Nucleic Acid Therapeutics |
| 11:45 | Dr Richard Scott, CEO, Genomics England | Advancing Individualised Medicine - Rare Therapies and Genomics |
| 12:00 | Dame June Raine DBE, CEO, Medicines and Healthcare products Regulatory Agency | Development of Therapies for Rare Diseases - The UK Regulator’s Role |
| 12:15 | Panel Discussion: Above speakers and Dr Kath Bainbridge, Head of Rare Diseases and Emerging Therapies, Department of Health and Social Care) and Melanie Dixon (Trustee, Cure DHDDS) | Chair: Dr Dan O'Connor |
| 13:00 | Lunch (1 hour) | |
| 14:00 | SESSION 3 Oxford-Harrington Rare Disease Centre | Chairs: Dr Diana Wetmore, Harrington Discovery Institute and Prof Carlo Rinaldi, University of Oxford |
| 14:05 | Prof Angela Russell, Professor of Medicinal Chemistry, University of Oxford | Next Generation Utrophin Modulators for Duchenne Muscular Dystrophy |
| 14:25 | Prof Haiyan Zhou, Professor in Genetic Therapy, University College London | Saving Sense by Antisense: Advancements in the UK and Beyond |
| 14:45 | Prof Loren Walensky, Professor of Pediatrics, Harvard University/Dana-Farber Cancer Institute | Stapled Peptide PROTACs: A 3-in-1 Drug Modality for Cancer Treatment |
| 15:05 | David Hipkiss, Executive Chair, AlveoGene | AlveoGene: Providing New Genetic Treatments for Respiratory Diseases |
| 15:25 | Coffee Break (30 mins) | |
| 15:55 | SESSION 4 Partnerships in Rare Disease Drug Development | Chair: Prof Seth Field, Harrington Discovery Institute |
| 16:00 | Prof Hugh Watkins, Radcliffe Professor of Medicine, University of Oxford | CureHeart: Developing Genetic Therapies for Inherited Heart Muscle Disease |
| 16:20 | Dr Fredrik Erlandsson, Chief Medical Officer, Purespring Therapeutics | Podocyte Targeted Gene Therapy to Treat Kidney Diseases |
| 16:40 | Dr Rena Baek, Senior Director, Rare Disease Research and Preclinical Development, Moderna Therapeutics | mRNA as a Potential Therapy for Rare Metabolic Disease |
| 17:00 | Dr James McArthur, President and CEO, PepGen | Leveraging the EDO Technology for the Treatment of Neuromuscular Diseases |
| 17:20 | Dr Vanessa Almendro, Vice President of Science and Technology, Head of Innovation, Danaher Corporation | When Traditional Approaches Fail: Reimagining the Drug Development Paradigm |
| 17:40 | Closing Remarks | |
| 17:45 | End of Session | |
| 18:00 | Drinks Reception | |
| 19:00 | Conference Dinner in Trinity Hall | |
Thursday 26th September | ||
|---|---|---|
| 8:30 | Registration and Coffee | |
| 8:55 | SESSION 5 Early Intervention for Neuromuscular Diseases | Chair: Prof Laurent Servais, University of Oxford |
| 9:00 | Prof Laurent Servais, Professor of Paediatric Neuromuscular Diseases, University of Oxford | From Genetic to Genomic Newborn Screening - How Early Identification Could Be Game Changing |
| 9:15 | Emily Reuben OBE, Co-founder and CEO, Duchenne UK | Early Diagnosis of DMD: Thoughts from Patients and Caregivers |
| 9:30 | Dr Ash Dugar, Chief Medical Affairs Officer, Dyne Therapeutics | Advancing Dyne’s FORCE Platform for Treatment of Neuromuscular Disorders to Deliver for Patients |
| 9:45 | Panel Discussion: Above speakers and Prof Francesco Muntoni (Professor of Paediatric Neurology, University College London) | Chair: Prof Laurent Servais |
| 10:30 | Coffee Break (30 mins) | |
| 11:00 | SESSION 6 Therapeutics for Rare and Childhood Cancers | Chair: Prof Louis Chesler, The Institute of Cancer Research |
| 11:05 | Prof Louis Chesler, Paediatric Solid Tumour Biology and Therapeutics Group Leader, The Institute of Cancer Research, London | An Approach to Targeting the Oncogenic Drivers of Aggressive Childhood Brain and Solid Tumours |
| 11:25 | Prof John Anderson, Professor of Experimental Paediatric Oncology, UCL | Developing Switchable CAR-T Platforms for Children’s Oncology |
| 11:45 | Prof Sarah Blagden, Professor of Experimental Oncology, University of Oxford | Precision Prevention Trials For Cancer Susceptibility Syndromes |
| 12:05 | Prof John Letterio, Professor, Department of Pediatrics, Case Comprehensive Cancer Centre, Case Western Reserve University | Academic-Industry Partnerships: Advancing New Drugs for Childhood Cancers in Cleveland |
| 12:25 | Prof Georg Holländer, Head of Department of Paediatrics, University of Oxford | Closing Remarks |
| 12:30 | Lunch and Departure | |