#USA #Cleveland #Rare Disease #Oxford-Harrington #Scholar Award

Oxford-Harrington Rare Disease Centre Announces 2025 Scholars Advancing Promising Treatments

In a significant announcement made on November 20, 2025, the Oxford-Harrington Rare Disease Centre (OHC) revealed the recipients of its prestigious 2025 Rare Disease Scholar Award. This award seeks to propel groundbreaking discoveries from academic labs into clinical settings, paving the way for innovative treatments for rare diseases that affect millions worldwide.

The 2025 cohort comprises ten distinguished scientists who have dedicated their research to tackling various rare and ultra-rare diseases. Each scholar will receive $100,000 in funding along with tailored advisory support aimed at navigating the complexities of drug and business development. This initiative not only provides financial resources but also opens pathways for scholars to secure up to $1 million in investment funds based on the specific needs of their research projects.

Rare diseases are often termed "orphan diseases" and are defined as conditions that affect a small percentage of the population, typically fewer than 200,000 people in the U.S. Despite their rarity, it is estimated that they impact approximately 500 million individuals globally, half of whom are children. Alarmingly, less than 5% of the 7,000 identified rare diseases currently have approved treatment options.

The OHC, established through a collaboration between the University of Oxford in the UK and the Harrington Discovery Institute at the University Hospitals in Cleveland, Ohio, is committed to accelerating the development of therapies for these conditions. "Behind every rare disease is a person, a family, and a story of resilience," said Lord Cameron, Chair of the OHC Advisory Council. He further emphasized the importance of combining academic excellence with philanthropy and entrepreneurial insights in the quest for transformative drug development.

In line with its mission, the OHC aims to facilitate the introduction of 40 new rare disease treatments within the next decade. The current cohort's research spans various fields, including gene therapies, RNA therapies, and small molecule drugs. The innovative approaches being explored involve treatments targeting neurological disorders, metabolic issues, and specific forms of cancer. By harnessing cutting-edge technologies like gene therapy and precision medicine, the OHC believes it can provide targeted solutions to many previously untreatable conditions.

Matthew Wood, MD, PhD, the Director and Chief Scientific Officer of OHC, voiced optimism regarding the initiative, noting the impressive advancements made by the first set of scholars. He stated, "We have seen remarkable progress with many projects from our inaugural class moving closer to clinical application. The new 2025 Scholar Awards will introduce ten additional OHC programs focused on overcoming some of the most challenging rare diseases through pioneering therapies."

The following researchers are the awardees of the 2025 Rare Disease Scholar Award:

• - Rachel Bailey, PhD (University of Texas Southwestern Medical Center, Dallas, TX, USA): Developing gene therapy for a rare neurodevelopmental disorder linked to the SLC13A5 citrate transporter.
• - Esther Becker, PhD (University of Oxford, Oxford, UK): Investigating new small molecules to inhibit dysfunctional nerve functions in spinocerebellar ataxia.
• - Joseph Buxbaum, PhD (Icahn School of Medicine at Mount Sinai, New York, NY, USA): Creating treatments for the Autism-Spectrum Neurodevelopmental ADNP syndrome using small molecule drugs.
• - Matthew Gentry, PhD (University of Florida, Gainesville, FL, USA): Developing advanced enzyme therapy aimed at neurological glycogen storage diseases.
• - Albert La Spada, MD, PhD (University of California, Irvine, CA, USA): Focusing on silencer RNA therapy to treat rare juvenile-onset ALS (ALS4).
• - Michael Lin, MD, PhD (Stanford University, Stanford, CA, USA): Engineering a therapeutic solution for rare brain cancers through genetic programming.
• - Pengfei Liu, PhD (Baylor College of Medicine, Houston, TX, USA): Pioneering nucleic acid therapeutics for the ultra-rare CAMK2B neurodevelopmental disorder.
• - David Segal, PhD (University of California, Davis, CA, USA): Contributing to nucleic acid drug treatments for the Autism-Spectrum ADNP syndrome.
• - Anthony Shum, MD (University of California, San Francisco, San Francisco, CA, USA): Developing gene therapy for the progressive multimodal COPA syndrome.
• - Mingshan Xue, PhD (Baylor College of Medicine, Houston, TX, USA): Innovating gene therapy for the catastrophic CASK-related neurodevelopmental disorder.

For those interested in engaging with the OHC, the next funding application period for the Rare Disease Scholar Award will open on January 14, 2026. Additional information can be obtained from their website at OxfordHarrington.org/funding.