Partnering For Progress: Funding The Future Of Rare Disease Research
A conversation with Matthew P. Anderson, MD, Ph.D., co-director, Oxford-Harrington Rare Disease Centre
Rare diseases impact 500 million people worldwide, yet 95% have no approved treatment — not for lack of science, but because most discoveries stall long before the clinic. The Oxford-Harrington Rare Disease Centre (OHC) hopes to change that. The OHC is a partnership between the University of Oxford and the Harrington Discovery Institute in Cleveland, Ohio, and its goal is to advance 40 drugs into clinical trials by 2034.
It identifies high-potential breakthroughs and funds the early steps that typically prevent progress. The OHC offers the Oxford-Harrington Rare Disease Scholar Award to 10 scientists each year who are developing gene, RNA, cell, and small molecule therapies in the OHC’s priority areas: rare neurological diseases, metabolic and developmental diseases, and childhood cancers.
In this Q&A, Life Science Connect’s Morgan Kohler caught up with Matthew Anderson to discuss the center’s research efforts in drug discovery.
Why did the OHC narrow its R&D focus to the aforementioned priority areas and what solutions can it propose?
These areas represent some of the most severe and under-resourced conditions in rare disease medicine. They often have a strong genetic basis, which aligns with the OHC’s expertise in genetic and genomic therapies. By focusing on rare neurological, metabolic, and developmental diseases, as well as pediatric cancers, we can leverage cutting-edge science, such as nucleic acid, gene, and cell therapies, to address conditions with high unmet need and clear potential for clinical impact. Our second cohort of Rare Disease Scholars reflects this focus, such as advancing projects to explore gene therapy to cure SLC13A5 citrate transporter disorder — an ultra-rare and devastating pediatric neurodevelopmental disorder — or using silencer RNA therapy to treat ALS4, which is a rare juvenile-onset amyotrophic lateral sclerosis (ALS) neuromuscular disease.
Unmet need persists because each rare disease affects a small patient population, which makes them less attractive for traditional commercial models. Scientific complexity and limited funding also slow progress. The OHC offers a unique transatlantic model that combines Oxford University’s research excellence with Harrington Discovery Institute’s proven track record in drug development and physician–scientist expertise. Through philanthropic and nonprofit investment, we de-risk early-stage projects, bridging the gap from academic discovery to clinical application. We don’t just provide funding but also provide each scientist with an advisory team with advanced drug and business development support.
What does the preclinical work at the OHC look like to achieve the progression of 40 disease treatments to clinical trials by 2034?
Preclinical work at the OHC is focused on rigorous target validation, proof-of-concept studies, and optimization of therapeutic candidates. For example, we support disease mechanism understanding studies to help determine the most effective therapeutic strategies and explore novel proof-of-concept studies, such as gene replacement or silencing, using in vitro and in vivo models.
Given that so many rare diseases are genetic, the OHC is pioneering “therapeutic genomics,” a scalable approach to genetic medicines that leverages proven delivery platforms and oligonucleotide chemistries. This allows us to swap in corrective genetic components without restarting development, saving time and cost. We also integrate technologies such as gene-editing, silencing, or upregulation (CRISPR, base or prime base editing, or use of RNA or oligonucleotide approaches). In addition, we are adapting AI-driven analytics to enhance precision, efficiency, and rare to common disease pathway insights to better understand opportunities. These methodologies are critical for accelerating timelines and improving predictability in preclinical research.
We’ve supported research, both financially and through our scholar advisory teams, to optimize formularies and establish safety and tolerability in experimental or computational model systems. The ultimate goal of our preclinical work is to generate robust data needed to advance discoveries into human clinical trials.
Can you discuss how the OHC plans to tackle the issue of translation after development?
Translation is often the biggest hurdle in rare disease research, and the OHC addresses it through a collaborative approach that brings together the best medical research and drug development expertise, highly passionate people, and strong links into a supportive drug research and development ecosystem. Our Rare Disease Scholar Award provides funding, project management, and commercial strategy to advance promising therapies. We also foster partnerships with biopharma, investors, and regulatory bodies to ensure that therapies move efficiently from lab to clinic.
AlveoGene is a company we created and funded in 2023 with Oxford Science Enterprises and Old College Capital in partnership with six leading scientists from the U.K. Respiratory Gene Therapy Consortium. The company is focused on transforming rare respiratory disease outcomes using an inhaled gene therapy and is a prime example of how we foster partnerships to advance new treatments for rare diseases.
What advice do you have for companies that are working on drug development for rare diseases?
Rare diseases often have small, highly specialized patient populations. It’s therefore particularly important to engage with the patient communities early, since they can provide insights into meaningful endpoints, acceptable risk-benefit profiles, and practical considerations such as the burden of participation.
In 2018, a young girl named Mila became the first person in the world to be treated with a medicine designed for and tested in one person — and developed in under a year. In her case, it was a race against time to find a treatment for her rare condition, Batten disease. Mila’s family was in close touch with a physician–scientist at an academic lab who developed this tailored therapeutic. So, for companies working on treatments for rare diseases, be sure to engage patients and families early in the therapeutic development process, as was the case with Mila’s academic team.
Collaboration across patients, regulators, industry, and academics is essential in this space. Collaboration is something the OHC seeks to foster, such as through the Rare Therapies Launch Pad in the U.K., in which the OHC is a founding member, to help build the processes and infrastructure needed to bridge the gap between patients and the science.
What advice do you have for those interested in your Oxford-Harrington Rare Disease Scholar Award?
The OHC’s Rare Disease Scholar Award seeks novel approaches to treat and cure rare diseases — so we’re looking for proposals that demonstrate rigorous science, innovation, and potential for clinical impact. Demonstrate how your work can move from discovery to a viable therapeutic candidate and how it would benefit from access to a team of experts in drug development, regulatory strategy, and commercialization. Be prepared to engage actively with mentors and embrace a milestone-driven approach to ensure your project advances toward clinical impact. Our next call for applications opens in January 2026.
About The Expert
Matthew P. Anderson, MD, Ph.D., is co-director of The Oxford-Harrington Rare Disease Centre. He is also a professor in the Department of Pathology at University Hospitals, Cleveland, and Case Western Reserve University, as well as a visiting professor at the University of Oxford.
Prior to joining the OHC, he served as vice president of research and preclinical development and head of the neuroscience therapeutic focus area at Regeneron Pharmaceuticals. For many years, he served as chief of neuropathology at Harvard Medical School.
Anderson received his MD and Ph.D. degrees from the University of Iowa College of Medicine. He completed postdoctoral research training at Massachusetts Institute of Technology.