Harrington Discovery Institute at University Hospitals

We’re thrilled to share that AlveoGene, an innovative company supported by the Oxford-Harrington Rare Disease Centre, has received FDA Rare Pediatric Disease Designation (RPDD) for AVG-002, its novel inhaled gene therapy for neonatal Surfactant Protein B (SP-B) deficiency—a life-threatening, ultra-rare genetic condition. This designation is a vital milestone in advancing treatments for rare respiratory diseases, providing hope for newborns and their families where no viable options currently exist. It also paves the way for a Priority Review Voucher, accelerating future approvals and patient access. Harrington Discovery Institute at University Hospitals is proud to partner with the University of Oxford in creating the Oxford-Harrington Rare Disease Centre, aiming to deliver cures for rare diseases. Learn more about AlveoGene’s cutting-edge work: www.alveogene.com #RareDisease #GeneTherapy #HarringtonDiscovery #Innovation

Lord Cameron, Chair of our Advisory Council, highlights on Sky News the Oxford-Harrington mission to accelerate therapeutics development for rare diseases, and the compelling motivations behind his involvement. See more here: https://lnkd.in/d8wsFYqb

The Oxford-Harrington Rare Disease Centre announces the inaugural class of 2024 OHC Rare Disease Scholar Award Recipients, an exceptional group of scientists advancing groundbreaking treatments for rare and ultra-rare diseases. Chaired by former UK Prime Minister David Cameron, the OHC is committed to transforming lives with innovative therapies spanning rare neurological diseases, metabolic and developmental diseases, and childhood cancers. Each scholar will receive critical funding and industry support to accelerate their work toward clinical impact. Congratulations to the 2024 OHC Scholars!

Congratulations to the ten recipients of the 2024 Oxford-Harrington Rare Disease Scholars Award! This grant is awarded by the Oxford-Harrington Rare Disease Centre a partnership between the University of Oxford and Harrington Discovery Institute at University Hospitals and includes funding and personalized therapeutics development support to help advance new treatments into the clinic. Learn more: https://bit.ly/3YBwiWE #rarediseases University Hospitals

The Oxford-Harrington Rare Disease Centre Announces Inaugural 2024 Rare Disease Scholar Award Recipients 10 scientists have been selected for OHC’s first annual award to advance academic discoveries into clinical practice for rare diseases Jonathan S. Stamler, MD, President of Harrington Discovery Institute, University Hospitals added: “By supporting each OHC Scholar with funding and an experienced therapeutics development team, we are maximising the potential of their discoveries to advance into the clinic. We have seen success with this approach at Harrington Discovery Institute and we are excited about replicating the model at the OHC. I look forward to seeing the progress of these innovative initiatives and sharing their advancements as the OHC continues to work towards providing scalable solutions to address the rare disease challenge globally.”

Following the announcement of his role as Chair of our Advisory Council, Lord Cameron highlights in this video the Oxford-Harrington Rare Disease Centre as a brilliant collaboration between two great institutions: the University of Oxford and Harrington Discovery Institute at University Hospitals , translating science into medicines that make a difference. Read more: https://bit.ly/3NxM1zv

Former UK Prime Minister David Cameron Joins Oxford-Harrington Rare Disease Centre as Chair of our Advisory Council. As Chair of the Oxford-Harrington Rare Disease Centre Advisory Council, Lord Cameron will lead the international efforts of the OHC in its philanthropic mission, build partnerships, expertise and networks, while also focusing on extending global awareness of the OHC and its objectives. Lord Cameron said: “Of the causes I have advocated, this one really is very personal. Setting up Genomics England and establishing a world-leading database with hundreds of thousands of genomes is one of my proudest achievements as Prime Minister. Yet, to be truly transformational, genomics requires the best of academia, life sciences, pharmaceutical companies, philanthropy and venture capital from around the world to come together. That is what the Oxford-Harrington Rare Disease Centre sets out to do, convening those key players and uniting around a bold mission: to deliver 40 new treatments for rare diseases in the next decade. “As a father I know all too well the impact of rare diseases. We miss our son Ivan, who had a rare neurological disease, every day – all these years on our loss is still so raw. But the steps we are taking now means that, in the not-too-distant future, families like ours will have hope. It is vital that we try to give it to them.”  

Press Release: Oxford-Harrington Rare Disease Centre Appoints Influential UK Leader in Health Innovation Policy, Baroness Nicola Blackwood, to its Advisory Council. The Oxford-Harrington Rare Disease Centre (‘OHC’), a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio, aimed at driving cutting-edge rare disease breakthroughs, announces the appointment of Baroness Nicola Blackwood to the OHC Advisory Council. In her position on OHC’s Advisory Council, Baroness Blackwood will contribute to advancing OHC’s mission to drive life-changing discoveries in rare disease, with a particular focus on addressing critical unmet needs in the UK. Learn more: https://bit.ly/3MFNCD3

Seeking Innovators in Medicine Call for Nominations: Harrington Prize for Innovation in Medicine Do you know a physician-scientist whose work is transforming the field of medicine? Nominate them for the Harrington Prize, an award that celebrates outstanding innovation and clinical potential. Prize Highlights: ·       $20,000 honorarium ·       Lecture at the AAP-ASCI-APSA Joint Meeting ·       Participation in the Harrington Discovery Institute Scientific Symposium ·       Essay publication in the Journal of Clinical Investigation Eligibility Requirements: ·       Must hold an MD or MD/PhD ·       No age or citizenship restrictions ·       Self-nominations are not allowed Nomination Deadline: September 16, 2024. Learn more:  https://bit.ly/4d2ZgD4 Let’s recognize those making groundbreaking strides in medicine! #HarringtonPrize #MedicalInnovation #ScientificAchievement

Today, we know more about the causes of diseases than ever before. Yet treatments and cures are lacking for more than 500 million people worldwide. According to the National Institutes of Health (NIH), 80 to 90% of research projects fail before reaching testing in humans. Pharmaceutical companies and venture capital investors are understandably discerning, favoring projects with proven results and replicability. The gap between promising scientific discoveries and new medicines has been widening for at least 20 years, during which funding for translational research – research that verifies therapeutic targets – has been dissipating. Strategic philanthropy can fill the gap by supporting efforts to achieve early-stage results where many projects tend to languish and lose attention because of investment risk. Philanthropy is playing a unique and critical role in supporting translational medicine aimed at advancing more candidates to drug development. Importantly, philanthropic institutions can encourage knowledge exchange and resource sharing between “siloed” academic, pharma industry, and healthcare institutions. The result of such collaboration builds the bridge to translate scientific breakthroughs into medical treatments that improve and save lives. Learn more: https://bit.ly/43UfPht