Harrington Discovery Institute at University Hospitals

AlveoGene, the first company supported by the Oxford-Harrington Rare Disease Centre, has announced the launch of the first inhaled lentiviral gene therapy trial for cystic fibrosis, marking an important validation of its InGenuITy® platform. This milestone highlights the early impact of our unique drug discovery and development model—led by Harrington Discovery Institute at University Hospitals and the University of Oxford, with Oxford Science Enterprises as a key partner—to advance novel treatments for rare diseases. Learn more: https://lnkd.in/du-3SnvQ #GeneTherapy #RareDisease #CysticFibrosis #InhaledTherapies #OxfordHarrington #Biotech #HarringtonDiscoveryInstitute #InnovationToImpact

The twelfth annual Harrington Prize for Innovation in Medicine has been awarded to Owen N. Witte, MD, Distinguished University Professor and President's Chair in Developmental Immunology, David Geffen School of Medicine at UCLA. The award recognizes his foundational discoveries of targeted therapies that have transformed modern cancer treatment. Dr. Witte is internationally known for his contributions to the understanding of human leukemias and immune disorders. His work revealed the critical role of enzymes called tyrosine kinases in human disease. Dr. Witte discovered one of the first tyrosine kinases, the ABL oncoprotein, showing that its activity is responsible for causing chronic myeloid leukemia (CML)—a cancer of white blood cells. He predicted that drugs that inhibit the tyrosine kinase would have therapeutic benefit. Based on Dr. Witte’s work, the drug imatinib, an inhibitor of tyrosine kinase ABL, was developed as frontline therapy. Imatinib increases the 8-year survival rate for CML from 6% to 87%. Dr. Witte subsequently discovered Bruton’s tyrosine kinase (BTK). He provided evidence that BTK’s tyrosine kinase activity was important for both normal immune function (loss of BTK led to immunodeficiency disease) and white blood cell cancers—ultimately spurring the development of the BTK inhibitor drug ibrutinib, now used to treat several types of lymphomas and leukemias. The Harrington Prize for Innovation in Medicine, established in 2014 by the Harrington Discovery Institute University Hospitals and the American Society for Clinical Investigation (ASCI), honors physician-scientists who have moved science forward with achievements notable for innovation, creativity and potential for clinical application. Learn more: https://bit.ly/4c2ax7r

Baroness Nicola Blackwood, Chair of Genomics England and member of the Oxford-Harrington Rare Disease Centre (OHC) Advisory Council, brings a unique perspective to rare disease research and policy. Diagnosed with Ehlers-Danlos Syndrome (EDS) after a decades-long diagnostic journey, she is deeply committed to improving outcomes for the 400+ million people affected by rare diseases globally. "Over 7,000 rare diseases affect nearly 400 million people worldwide, yet only 5% have an approved treatment," she says. "That is why the OHC’s mission to deliver 40 new rare disease medicines in the next decade is so critical.” Learn more: https://lnkd.in/g5NR_5hY

The Oxford-Harrington Rare Disease Centre Rare Disease Scholar Award Call is closing soon. This award supports MD and PhD researchers in the UK, US, and Canada to advance promising discoveries from academic labs into clinical practice. Successful applicants will receive £100,000 (UK) or $100,000 (US/Canada) in grant funding, one year of therapeutics development support with potential renewal, access to advanced research facilities, and opportunities for additional acceleration and investment funds. Applications close on 10th March 2025. For more information and to apply, visit: https://lnkd.in/dXQHfEN6

Can personalized medicine change the future of rare disease treatment? Dr. Timothy Yu, a 2024 Oxford-Harrington Rare Disease Centre Rare Disease Scholar, is pioneering individualized genetic therapy for PEX1 Zellweger Syndrome—a devastating neurometabolic disorder with no cure. His work with the OHC is advancing antisense oligonucleotide (ASO) therapy to correct genetic mutations at the source, offering new hope for patients. Through groundbreaking research, the OHC is driving bold science forward to bring life-changing treatments to rare disease patients. Learn more: https://bit.ly/41eFnWN #RareDiseaseDay #GenomicMedicine

A new frontier in cancer treatment. Dr. Charles Gersbach, a 2024 Oxford-Harrington Rare Disease Centre Rare Disease Scholar, is developing an epigenetically enhanced CAR T cell therapy for Hodgkin’s Lymphoma—a rare blood cancer. With support from the OHC, his work aims to reprogram immune cells for greater durability and effectiveness, offering new hope to patients with limited treatment options. By accelerating life-changing science into medicines, the OHC is uniting researchers, clinicians, and industry leaders to drive the delivery of new rare disease treatments. Learn more: https://bit.ly/3WZkfBg #RareDiseaseDay #DrugDiscovery

Breakthrough treatments for rare diseases start with bold ideas. Dr. Bowen Li, a 2024 Oxford-Harrington Rare Disease Centre Rare Disease Scholar, is pioneering a new approach to treating cystic fibrosis using lipid nanoparticles to precisely deliver therapeutic RNA to disease-causing cells. His research, supported by the Oxford-Harrington Rare Disease Centre, could transform treatment for patients with few therapeutic options. “The beauty of the lipid nanoparticles is that we can very precisely deliver drugs directly into the cells that cause the diseases. By leveraging lipid nanoparticles as delivery vehicles, we aim to deliver therapeutic RNA molecules that can overcome cellular genetic mutations. This therapeutic approach aligns perfectly with the needs of rare disease populations, offering hope for individuals suffering from conditions that currently lack effective treatments,” Dr. Li said. Learn more about Dr. Li’s work and the OHC mission: https://bit.ly/3CNeRKI #RareDiseaseDay #DrugDiscovery

This Rare Disease Day, the Oxford-Harrington Rare Disease Centre joined the Global Chain of Lights to raise awareness for the nearly half a billion people worldwide living with a rare disease. This year, we illuminated the Magdalen College, University of Oxford Chapel Wall with a display in the Rare Disease Day colours. This illumination is only a part of our mission to raise awareness and advance treatments for rare disease patients. By accelerating breakthrough therapies through the Oxford-Harrington Rare Disease Centre, we’re bringing hope to millions.

95% of rare diseases have no treatment. This Rare Disease Day, we’re working to change that. At the Oxford-Harrington Rare Disease Centre, we are on a mission to develop 40 new therapies by 2034. By combining Oxford’s world-leading medical research with the drug development expertise of Harrington Discovery Institute at University Hospitals we are accelerating the discovery of life-changing treatments for rare disease patients. Through our Therapeutics Accelerator and the Oxford-Harrington Rare Disease Scholar Award, we are advancing innovative treatments from early discovery through to clinical trials. On Rare Disease Day, we stand with rare disease patients, researchers, and advocates to push for innovation, better care, and real solutions. Watch our new video to see how we’re driving global impact, together with Nicola Blackwood and David Cameron. Learn more: https://lnkd.in/dhwqTKXh #RareDiseaseDay #RareDiseases #RareDiseaseDay2025 #ResearchForRare #CareForRare

We are pleased to see the latest episode of the #StrategyandInsider podcast spotlighting the work of the Oxford-Harrington Rare Disease Centre (OHC). In this insightful conversation with Dr. Thomas Solbach, OHC Director and Chief Scientific Officer, Professor Wood, shares his journey in neuroscience, the challenges of translating academic research into real-world solutions, and the ambitious mission of the OHC to bring 40 new drugs into clinical trials over the next decade. The OHC, a partnership between the University of Oxford and Harrington Discovery Institute at University Hospitals is driving groundbreaking progress in the fight against rare diseases. Tune in to the episode to learn more about the power of scientific collaboration: https://bit.ly/4hPtOLq #RareDisease #MedicalInnovation