Oxford-Harrington Rare Disease Centre Symposium 2024 Video Highlights

November 11, 2024

The Oxford-Harrington Rare Disease Centre (‘OHC’) hosted its biennial Symposium on 24th-26th September 2024 at Trinity College, University of Oxford. The event brought together leaders from academia, biotech, pharma, venture capital, philanthropy, and patient groups to connect, engage and inform the rare disease community.  

The Oxford-Harrington Rare Disease Symposium 2024 aimed to explore how cutting-edge research, innovative treatment strategies, and collaboration can drive the development of effective therapies for rare diseases. 

The three-day event was structured around six scientific sessions that covered topics, such as therapeutic genomics, the Rare Therapies Launch Pad, early interventions for neuromuscular diseases, rare cancers, and partnerships in rare disease drug development. 

Professor Matthew Wood, Director and Chief Scientific Officer of the OHC, said: "We have a mission, which is to develop new medicines for rare diseases, and our goal over the next ten years is to deliver at least forty new drugs to clinical trials, which we hope would benefit many millions of patients suffering from rare diseases. That is why this symposium, which is really focused on the development of new therapies, is so important to us." 

The opening session of the 2024 Symposium included a talk from Baroness Nicola Blackwood, Chair of Genomics England and member of OHC’s Advisory Council, who spoke about genomic innovation in the United Kingdom.

Baroness Nicola Blackwood said: "By leveraging the strengths of the UK's genomic and life sciences ecosystem, where we have established so much groundwork and so much expertise already, the ultimate goal here is to create a new scalable and sustainable pathway from early diagnosis to treatment, monitoring and reimbursement that will then be applicable for all appropriate patients." 

The Opening Session also included talks from Julia Vitarello, CEO of Mila’s Miracle Foundation, and Professor Fyodor Urnov, UC Berkeley. The session concluded with an overview of the 'Harrington Model and Accelerator' from Dr Jonathan Stamler, Harrington Discovery Institute.

Session Highlights 

Opening Session

• Generation Genome: Integrating Rare Therapies into Healthcare, Baroness Nicola Blackwood, Chair, Genomics England
• CRISPR Cures On-Demand at Nationwide Scale: an Actionable Path, Professor Fyodor Urnov, UC Berkeley.
• From Mila to Millions, Julia Vitarello, CEO of Mila’s Miracle Foundation

Therapeutic Genomics 

• Therapeutic Genomics - Learning to Treat Rare Genetic Disorders at Scale, Professor Stephan Sanders, University of Oxford
• De Novo Variants in the Non-coding snRNA RNU4-2 Cause a Frequent Neurodevelopmental Syndrome, Professor Nicky Whiffin, University of Oxford
• Developing Therapeutic Genomics Approaches For Immunohaematology, Professor James Davies, University of Oxford 

Rare Therapies Launch Pad 

• Scientific, Regulatory, and Ethical Implications of Precision Nucleic Acid Therapeutics, Professor Timothy Yu, Harvard University 

Oxford-Harrington Rare Disease Centre 

• Saving Sense by Antisense: Advancements in the UK and Beyond, Professor Haiyan Zhou, University College London
• AlveoGene: Providing New Genetic Treatments for Respiratory Diseases, David Hipkiss, AlveoGene 

Partnerships in Rare Disease Drug Development 

• CureHeart: Developing Genetic Therapies for Inherited Heart Muscle Disease, Professor Hugh Watkins, University of Oxford 
• When Traditional Approaches Fail: Reimagining the Drug Development Paradigm, Dr Vanessa Almendro, Danaher Corporation 

Early Intervention for Neuromuscular Diseases 

• From Genetic to Genomic Newborn Screening - How Early Identification Could Be Game-Changing, Professor Laurent Servais, University of Oxford
• Early Diagnosis of DMD: Thoughts from Patients and Caregivers, Emily Reuben OBE, Duchenne UK

Therapeutics for Rare and Childhood Cancers 

• An Approach to Targeting the Oncogenic Drivers of Aggressive Childhood Brain and Solid Tumours, Professor Louis Chesler, Institute of Cancer Research
• Precision Prevention Trials For Cancer Susceptibility Syndromes, Professor Sarah Blagden, University of Oxford
• Academic-Industry Partnerships: Advancing New Drugs for Childhood Cancers in Cleveland, Professor John Letterio, University Hospitals Rainbow Babies & Children’s Hospital 

About Oxford-Harrington Rare Disease Centre 

The Oxford-Harrington Rare Disease Centre (OHC) is a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, in Cleveland, Ohio, US. The OHC combines world-leading strengths in research and therapeutics development from across these organisations to set the science and innovation agenda and drive cutting-edge rare disease breakthroughs to address the unmet need in rare diseases across the globe to deliver major clinical impact for patients. For more information, visit: OxfordHarrington.org | LinkedIn | X