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Advancing Rare Disease Research: 2025 Rare Disease Day

Posted on March 25, 2025 in Center for Rare Diseases

In honor of Rare Disease Day 2025, the Oxford-Harrington Rare Disease Centre (OHC), a collaboration between Harrington Discovery Institute at University Hospitals and the University of Oxford, continues to make significant strides in advancing therapies for rare diseases. The OHC is committed to bringing 40 new drugs into clinical trials by 2034.

Matthew P. Anderson, MD, PhD, Investigator at Harrington Discovery Institute and Co-Director of the OHC, recently presented at the Cleveland National Organization for Rare Disorders (NORD) Rare Disease Day event. The event, attended by patients, families, and physicians/physician-scientists and streamed virtually, provided Dr. Anderson an opportunity to share the OHC’s ongoing efforts in advancing rare disease treatments. One key development featured was the inaugural 2024 OHC Scholars, as well as progress on AlveoGene’s AVG-002, a novel inhaled gene therapy for alpha-1 antitrypsin deficiency disease (AATD), a degenerative condition that can lead to early onset emphysema.

The OHC remains committed to transforming the treatment landscape for rare diseases through strategic partnerships and cutting-edge research. This work not only addresses the pressing needs of patients with rare conditions but also advances innovative therapies that could reshape the future of rare disease treatment.

Harrington Discovery Institute and the University of Oxford continue to make meaningful progress toward improving the lives of rare disease patients, driven by collaboration, innovation, and a shared mission to bring life-changing therapies to market.

Advancing Rare Disease Research: 2025 Rare Disease Day

Dr. Anderson participated in a clinical expert panel discussion at the Cleveland National Organization for Rare Disorders (NORD) Rare Disease Day event.

Advancing Rare Disease Research: 2025 Rare Disease Day

Dr. Anderson presented at the Cleveland National Organization for Rare Disorders (NORD) Rare Disease Day event.

Watch 2025 NORD Rare Disease Day

Learn More about the Oxford-Harrington Rare Disease Centre

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