Online Order Get In Touch
CN JP KR

What would you like to find?

Oxford-Harrington Rare Disease Centre Announces 2025 Scholar Awards to Accelerate Gene and RNA Therapies

Nov 20 , 2025
share:

OXFORD, UK and CLEVELAND, Ohio – November 20, 2025 — The Oxford-Harrington Rare Disease Centre (OHC), a unique partnership between the University of Oxford and the Harrington Discovery Institute at University Hospitals, Cleveland, today announced the recipients of its 2025 Rare Disease Scholar Award. The awards are designed to move promising academic discoveries into clinical practice to address the estimated 7,000 known rare diseases, fewer than 5% of which have approved treatments. A number of the gene therapy projects rely on adeno-associated virus (AAV) vectors for delivery.

Accelerating New Treatments with Funding and Expertise

Each of the ten new OHC Scholars will receive $100,000 (or £100,000) in initial grant funding, along with a dedicated advisory team to provide advanced drug and business development support. Scholars will also have the opportunity to compete for additional acceleration funds and qualifying investment funds up to $1,000,000.

“At OHC, we believe that by bringing together the best of academia and cutting-edge medical research… we can achieve transformational results, closing the gap between research and drug development and turning hope into the reality of new treatments,” said Lord Cameron, Chair of the OHC Advisory Council.

The OHC has set an ambitious mission to advance 40 new treatments into clinical trials by 2034.

2025 Cohort Focuses on Cutting-Edge Genetic and Cellular Approaches, Including AAV

The 2025 cohort is tackling a wide range of rare conditions affecting the brain, immune system, metabolism, and cancer, utilizing pioneering techniques such as AAV gene therapy, RNA therapy, enzyme replacement, and targeted small molecules.

“With the 2025 Scholar Awards, we are adding ten new OHC programs and tackling some of the most challenging rare diseases through pioneering gene, RNA, small molecule and cell-based therapies,” said Matthew Wood, MD, PhD, Director and Chief Scientific Officer of the OHC. The deployment of AAV technology is integral to the success of several of these programs.

Full List of 2025 OHC Rare Disease Scholar Award Recipients

 

Recipient Institution Field of Research / Approach Target Disease
Rachel Bailey, PhD University of Texas Southwestern Medical Center, Dallas, TX, USA AAV Gene Therapy Ultra-Rare, Devastating Neurodevelopmental Metabolic Disorder of the SLC13A5 Citrate Transporter
Esther Becker, PhD University of Oxford, Oxford, UK New Small Molecules to Inhibit Abnormal Nerve Function Preserve and Restore Muscle Function in Spinocerebellar Ataxia
Joseph Buxbaum, PhD Icahn School of Medicine at Mount Sinai, New York, NY, USA Small Molecule Drugs Treatment of the Autism-Spectrum Neurodevelopmental ADNP Syndrome
Matthew Gentry, PhD University of Florida, Gainesville, FL, USA Advanced Brain Penetrant Enzyme Therapy Treat Neurological Glycogen Storage Diseases
Albert La Spada, MD, PhD University of California, Irvine, Irvine, CA, USA Silencer RNA Therapy Rare, Juvenile-onset ALS Neuromuscular Disease, ALS4
Michael Lin, MD, PhD Stanford University, Stanford, CA, USA Genetically Programmed Therapeutic for Sensing and Killing Rare Brain Cancers
Pengfei Liu, PhD Baylor College of Medicine, Houston, TX, USA Precision Nucleic Acid Therapeutics Ultra-Rare CAMK2B Neurodevelopmental Disorder
David Segal, PhD University of California, Davis, Davis, CA, USA Nucleic Acid Drugs Treatment of the Autism-Spectrum Neurodevelopmental ADNP Syndrome
Anthony Shum, MD University of California, San Francisco, San Francisco, CA, USA Gene Therapy Progressive, Debilitating, Multisystem COPA Syndrome
Mingshan Xue, PhD Baylor College of Medicine, Houston, TX, USA Gene Therapy Catastrophic CASK-Related Neurodevelopmental Disorder

 

Source:

https://news.uhhospitals.org/news-releases/articles/2025/11/oxford-harrington-rare-disease-centre-announces-2025-scholars-advancing-promising-treatments

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Previous Next

Related Services

Download

Login

Don't have an account? Please register
Account*
Password*
Code*
Refresh
Forgot password?
Logging in indicates that you have read and accepted the Registration Agreement and User Agreement
Log in with other accounts

New User Registration

Already have an account?
First Name*
Middle Name
Last Name*
Organization*
Organization Type*
Country/State*
Email Address*
Set Password*
Confirm password*
Refferal Code*

Reset Password

Return to
Email*
Code*
New password*
Confirm password*

Google Account Binding

Organization*
Organization Type*
Country/State*