2024 Rare Disease Day Webinar

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Pioneering a Path to Rare Disease Therapeutics

In recognition of Rare Disease Day 2024, the Oxford-Harrington Rare Disease Centre (OHC) brought together a distinguished panel of experts for its 4th annual Rare Disease Day Webinar on February 28. This year’s webinar, "Pioneering a Path to Rare Disease Therapeutics," illuminated groundbreaking initiatives and insights aimed at addressing the unmet needs of rare diseases.

Guest speakers shared their expertise and perspectives on various facets of rare disease therapeutics:

• Eric Alton, Professor, Imperial College London: As the Coordinator of The UK Respiratory Gene Therapy Consortium, Professor Alton elucidated his team's collaborative efforts with new investors to deploy innovative approaches in delivering effective inhaled gene therapies to patients.
• Nicola Blackwood, Chair, Genomics England: Baroness Blackwood, a prominent figure in health innovation policy, shared her personal journey living with Ehlers-Danlos Syndrome, underscoring the importance of patient advocacy and empowerment in rare disease management.
• Claire Brown, PhD, Partner, Life Science at Oxford Science Enterprise: Dr. Brown shed light on AlveoGene's trajectory, having been intricately involved in the company's inception and initial investment round, highlighting the pivotal role of partnerships in driving therapeutic innovation.
• David Hipkiss, Executive Chair, AlveoGene: Mr. Hipkiss delved into AlveoGene, OHC Therapeutics Accelerator’s inaugural investment venture, dedicated to revolutionizing outcomes for rare respiratory diseases through inhaled gene therapy.
• Jonathan Stamler, MD, President and Co-Founder of Harrington Discovery Institute: Dr. Stamler provided updates on OHC's vision and global initiatives aimed at advancing cures for rare diseases, emphasizing the imperative of collaborative research and translational efforts.
• Matthew Wood, Professor, University of Oxford and Director and Chief Scientific Officer, Oxford-Harrington Rare Disease Centre: Professor Wood articulated OHC's mission, vision, and recent milestones, outlining the center's commitment to catalyzing transformative breakthroughs in rare disease therapeutics.
• Diana Wetmore, PhD, Vice President of Harrington Discovery Institute's Therapeutics Development Center: Dr. Wetmore offered insights into Harrington Discovery Institute's experience in advancing rare disease treatments, underscoring the significance of fostering an ecosystem conducive to therapeutic innovation.

Unveiling the Pioneering Therapeutics Accelerator

Central to the webinar's discourse was the launch of the Therapeutics Accelerator, a groundbreaking initiative by the Oxford-Harrington Rare Disease Centre poised to revolutionize the landscape of rare disease therapeutics. The Accelerator aims to identify, fund, and advance innovative projects from academic partners in the UK and the US, with the ambitious goal of ushering 40 new potentially life-changing therapies into clinical trials over the next decade, targeting multiple approvals in key markets.

Operationalizing a unique non-profit/for-profit model, the Accelerator pledges to commit up to £200 million, bolstered by support from a strategic partnership between University Hospitals in Cleveland, Ohio, University of Oxford, and investment company Oxford Science Enterprises. Former UK Prime Minister David Cameron is set to lead the international endeavors of the Accelerator as Founding Chair of the Oxford-Harrington Rare Disease Centre Advisory Council, underscoring the initiative's global significance and commitment to transformative change.

Join Us in Our Mission to Deliver Cures for Rare Diseases

The Oxford-Harrington Rare Disease Centre's 4th annual Rare Disease Day Webinar not only provided a platform for insightful discourse and collaboration but also heralded a new era of innovation and hope in the quest to combat rare diseases. With the launch of the pioneering Therapeutics Accelerator, the Centre reaffirms its unwavering commitment to driving tangible impact and ushering in a future where no disease is too rare to conquer.

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