Oxford-Harrington Rare Disease Centre
Partnership of the University of Oxford and Harrington Discovery Institute. Combining expertise in discovery science and therapeutics development to accelerate cures for rare diseases.
Visit us at: www.oxfordharrington.org
- The next VITALS session will feature Matthew Anderson, MD, PhD, a global leader in rare disease innovation and translational science. He is the co-director of the Oxford-Harrington Rare Disease Centre, with groundbreaking work spanning academia and industry. Register: tinyurl.com/f534nvw4
- The Oxford-Harrington Rare Disease Centre invites proposals for its 2026 Rare Disease Scholar Award, supporting the development of innovative therapies for rare diseases. Submissions close on March 9, 2026. Learn more: tinyurl.com/3ynf6xt7
- In a recent episode of RARECast, Matthew Wood, Director and CSO of the OHC, discusses the challenges of rare disease drug development, the resources the OHC brings to address them, and the mechanisms established to help accelerate therapeutic development. Listen now: art19.com/shows/rareca...
- In a recent episode of the In Vivo podcast, Matthew Wood discusses why rare disease research and drug development are at a pivotal moment and what must change to unlock progress for the ~500 million people impacted by rare diseases. 🎧Listen now: shorturl.at/y88MR #RareDiseases #DrugDevelopment
- As the year draws to a close, we are grateful to our Scholars, partners and Advisory Council members for their continued commitment to advancing treatments and cures for rare diseases.
- Thank you for helping drive discovery, collaboration and impact in 2025. We look forward to building on this momentum together in the year ahead.
- Last week, the Oxford-Harrington Rare Disease Centre convened its Advisory Council in Oxford to reflect on a year of progress and to set priorities for 2026. Read more: tinyurl.com/5c2kwcny
- Chaired by David Cameron, discussions highlighted advances in rare disease research, a Scholar spotlight from Professor Angela Russell, and insights from Genomics England on how genomic science is accelerating discovery and informing new therapeutic pathways for rare diseases.
- Council members also met with partners at Oxford Science Enterprises and, later, with the University of Oxford’s Vice-Chancellor, Professor Irene Tracey, during a visit to the Schwarzman Centre for the Humanities.
- A special seminar on the human impact of ultra-rare diseases—and the growing momentum behind Cure DHDDS —takes place next Tuesday, 2 Dec (2:30–3:30pm) at the Botnar Research Centre, Oxford. 🔹 Melanie Dixon (CureDHDDS) 🔹 Prof. Emyr Lloyd-Evans (Cardiff University) 🔗 talks.ox.ac.uk/talks/id/b90...
- Today, we are pleased to announce the 10 outstanding scientists selected for the 2025 Rare Disease Scholar Award, who will drive scientific breakthroughs to transform treatment options for the 500M people worldwide affected by rare diseases. Read more: tinyurl.com/49baysns
- Earlier in November, the OHC team joined the programme and conversations at the CamRare Summit 2025, exploring how scientific advances, policy shifts and new models of collaboration could shape the next decade of rare disease research and care. Photos: Jeremy Peters #RAREsummit25
- Earlier this week, Prof Matthew Wood, OHC Director, spoke at the FT Live Global Pharma & Biotech Summit. He said: "It was valuable to highlight the essential role of academic drug development in addressing rare diseases and the importance of regulatory innovation."
- Yesterday, we heard from Professor Carlo Rinaldi, Neurological Disorders Theme Lead at the Oxford-Harrington Rare Disease Centre, who spoke at the Cambridge Rare Disease Network (CamRARE) Rare Summit 2025 about the next decade of rare disease innovation.
- MHRA reforms mark a vital step toward faster, safer innovation for the 500M people worldwide living with rare diseases. A more adaptive, evidence-based framework will help researchers and clinicians deliver life-changing therapies faster. Read more: tinyurl.com/msurx5ju
- Every breakthrough starts with a bold idea—and the support to make it real. Join the Oxford-Harrington Rare Disease Centre webinar to hear how scientists & philanthropists are working together to advance cures for the world’s rarest diseases. 🔗 www.harringtondiscovery.org/events
- Professor Matthew Wood, Director of the Oxford-Harrington Centre, joins the FT Live Global Pharma and Biotech Summit to explore the future of healthcare innovation. 📅 11–12 Nov | London & online 🔗 bit.ly/4pPcJpd #FTPharma #Biotech
- Join Oxford-Harrington Scholars Angela Russell and Michael Pacold as they share advances in Duchenne muscular dystrophy and HDPL Encephalopathy in our upcoming webinars on November 5th and 13th. Register now: www.harringtondiscovery.org/events #OxfordHarrington #RareDisease
- On 14 Oct at the Francis Crick Institute, the #RareSummit2025 will unite leaders in science, healthcare & policy to accelerate diagnosis & treatment for rare diseases. 🔗 Register: tinyurl.com/4xe26ppm #AccelerateRare
- On #InternationalAtaxiaAwarenessDay, we spotlight progress in ARSACS research. In Oxford, Professor Esther Becker is using patient-derived cerebellar organoids to uncover why brain cells are vulnerable and to open new paths toward treatment. 🔗 tinyurl.com/ye6k9cx4 #RareDisease #AtaxiaAwareness
- Today, OHC Co-Director Dr Matthew Anderson gave a talk hosted by Prof Carlo Rinaldi, at the Institute of Developmental and Regenerative Medicine - University of Oxford, on how autism genetics provides a window into the neuronal circuits that regulate human behaviour.
- September is #ChildhoodCancerAwarenessMonth, a reminder of the urgent need for new treatments.
- Professor Louis Chesler, 2024 OHC Rare Disease Scholar, is developing regulatable CAR-T therapies that could transform treatment for children with brain tumours.
- His approach equips CAR-T cells with molecular “switches” — offering doctors precise control and new hope for children with cancer.
- Read more: www.oxfordharrington.org/events-news/...
- Oxford researchers are invited to a seminar with Dr Matthew Anderson on "Neural Circuit Targets of Behavioral Medicines: Insights Through Autism Spectrum Disorder Genetics." 📅 19 Sept | 11:45–12:45 📍 IDRM, Old Road Campus 🔗 Register for free: talks.ox.ac.uk/talks/id/afb...
- On #DuchenneAwarenessDay, we celebrate the power of patient advocacy. When Alex Johnson OBE and Emily Reuben OBE founded Duchenne UK, their shared mission was clear: to end Duchenne muscular dystrophy. In just over a decade, their work has transformed care across the UK. 🔗 tinyurl.com/ynz7anm5
- As Sarepta's gene therapy clinical trials for limb-girdle muscular dystrophy (LGMD) have been placed on hold, many patient families have been left without alternative treatment options. Read more in this article by Jason Mast: shorturl.at/zBQmI
- On 14 Nov 2025, Dravet Syndrome UK will host the #DSUKConference2025 in London St Pancras & online 🩺 The UK’s leading Dravet-focused scientific meeting will share research and clinical updates for healthcare professionals. Agenda & booking: bit.ly/DSUKProfConf25
- Professor Carlo Rinaldi, one of ten 2025 Oxford-Harrington Rare Disease Scholar Award recipients, is developing a novel RNA-based therapeutic strategy for Spinal and Bulbar Muscular Atrophy (SBMA), a rare and currently untreatable neuromuscular disorder. Read more: tinyurl.com/d37tvuxb
- To stay up to date with news from the OHC and the wider rare disease community, sign up for our quarterly newsletter. The OHC newsletter contains updates on our latest projects, funding announcements, events, and initiatives. Sign up now: oxfordharrington.us17.list-manage.com/subscribe?u=...
- At just 4 weeks old, Albert was diagnosed with a rare neonatal epilepsy, thanks to rapid genetic testing. His mum shares how a precise diagnosis gave Albert a fighting chance, and why more progress is still needed. Read more: shorturl.at/pKH6o Support Albert: shorturl.at/rAI6v #RareDisease
- Professor Michele Jacob, one of ten 2024 Oxford-Harrington Rare Disease Scholars, is developing the first targeted treatment for CTNNB1 syndrome, a rare genetic disorder with no current therapies. Read more about the project:🔗 lnkd.in/gntnHenF
- Despite promising scientific advances into new therapies, many rare and ultra-rare diseases remain without treatments due to lack of funding for clinical development and trials. Link to the full article: shorturl.at/pvyoC
- One such example is the disease caused by a mutation in a gene called DHDDS. Although fewer than 100 people in the world are known to have this mutation, research into treatments for it has the potential to benefit wider numbers of people.
- This is why patient advocates Dominic Nutt and Alexander Masters are developing new routes for funding trials that could help families, such as Cure DHDDS's trustee Melanie Dixon and two of her children, Tom and Rosie, who have a DHDDS genetic mutation.
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View full threadAlthough still in development, this new approach has the potential to finance a number of rare disease trials and advance treatments that might otherwise remain out of reach.
- We’re delighted to announce that Dr Antonio García Guerra has received the first FA Alliance Catalyst Fund award. His project will develop a non-viral gene editing platform to correct the frataxin gene in cardiac muscle cells, advancing urgently needed therapeutic strategies for FA.
- Supported by Oxford’s Translational Research Office in combination with philanthropic funding, this award is part of a broader FA Alliance effort to accelerate research into new treatments.
- Are breakthrough therapies for ultra-rare diseases within reach? At the 2025 #PrixGalienUKForum, OHC Director Prof Matthew Wood joined global leaders to discuss the future of individualised medicine. Watch the panel chaired by Dame June Raine: lnkd.in/dP8_zbAE
- 📢Applications are still open for the FA Alliance Innovation Fund. Up to £100k available for bold, high-impact R&D projects in Friedreich’s Ataxia. Open to Oxford researchers. Deadline: 18 June 2025 More info: shorturl.at/i5BwY 🔗
- Cystic fibrosis affects 105,000 people across 94 countries and is one of the most common life-threatening rare diseases worldwide. An OHC research project is developing an innovative approach to treat this genetic disease and improve patient outcomes. #CysticFibrosis #RareDisease
- Dr. Bowen Li, one of ten 2024 Oxford-Harrington Rare Disease Scholars, is developing a promising RNA-based therapy, which aims to address the genetic root cause of cystic fibrosis. #CysticFibrosis
- Dr. Li's research uses RNA molecules to restore the functionality of the CFTR protein and targets the root cause of the disease. This could become a more effective treatment option, particularly for patients who do not benefit from existing therapies. #CFAwarenessMonth
- Dr. Li's project benefits from the Oxford-Harrington Rare Disease Scholar Award, which combines funding and expert therapeutics development support to advance promising discoveries from academic labs into clinical practice and treatments for patients.
- At last week’s Harrington Discovery Institute Scientific Symposium, OHC Advisory Council Chair, Former UK Prime Minister David Cameron, joined global scientific leaders to spotlight progress in drug development for a variety of conditions, including rare diseases. Read more: shorturl.at/ri7QU
- This Saturday was #FAAwarenessDay — a powerful reminder of the urgent need for progress in #FriedreichsAtaxia research. Oxford researchers can now apply for the FA Alliance Innovation Fund until 18 June, 2025. 🔗 lnkd.in/dCDa4eP3 #CureFA
- Dr Bowen Li, Assistant Professor at the University of Toronto and 2024 Oxford-Harrington Rare Disease Scholar, is developing RNA-based therapies for #CysticFibrosis. Read more: shorturl.at/3MOZS
- FUNDING ALERT: The FA Alliance Innovation Fund is open for applications!🚨Do you have an innovative research hypothesis to test in Friedreich’s Ataxia? Apply now for funding up to £100k by 18 June 2025. 📩 Email: oxfordharrington@paediatrics.ox.ac.uk 🌐 More info: shorturl.at/tpIfY
