Event
Oxford-Harrington Rare Disease Centre Symposium 2024
24th - 26th September, 2024
The Oxford-Harrington Rare Disease Centre Symposium took place on 24-26 September 2024 at Trinity College, Oxford.
- 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 | |