Oxford-Harrington Rare Disease Centre
Research Services
Partnership of the University of Oxford and Harrington Discovery Institute
About us
Rare Disease Centre at the University of Oxford and Harrington Discovery Institute. Combining expertise in discovery science and therapeutics development to accelerate cures for rare diseases
- Website
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www.oxfordharrington.org
External link for Oxford-Harrington Rare Disease Centre
- Industry
- Research Services
- Company size
- 201-500 employees
- Headquarters
- Oxford
- Type
- Nonprofit
Locations
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Primary
Get directions
Oxford , GB
Employees at Oxford-Harrington Rare Disease Centre
Updates
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Oxford-Harrington Rare Disease Centre reposted this
Experienced C-Suite Level Life Science Professional, Chair, Board Member and Serial Entrepreneur
An excellent paper that continues to underscore the power of AlveoGene's InGenuITy®️ inhaled gene therapy platform. 2025 will be a pivotal year for AlveoGene as they seek to transform rare respiratory disease outcomes and provide lasting life-saving solutions where there are none
Our manuscript “Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial” has just been fully published in American Journal of Respiratory and Critical Care Medicine (https://lnkd.in/eG377qS7).
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The Oxford-Harrington Rare Disease Symposium 2024 brought together leaders from academia, biopharma, funders, patient organisations, and regulators to discuss the latest research and innovation shaping the future of rare disease therapeutics. The programme covered key topics in rare diseases, including therapeutic genomics, drug development exemplars, lifelong therapies for neuromuscular diseases and paediatric cancer. Sixteen of the presentations, led by international leaders in the field, are now available online. Watch them here: https://lnkd.in/dmVCGHrS
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The Oxford-Harrington Rare Disease Centre will partner with a new MRC Centre of Research Excellence (CoRE) in Therapeutic Genomics, directed by Professor Stephan Sanders, alongside co-director Professor Deborah Gill (University of Oxford), and Chemistry Nobel Prize winner Jennifer Doudna (founder of the Innovative Genomics Institute). The new MRC CoRE in Therapeutic Genomics aims to transform the diagnosis and treatment of genetic disorders by enabling the mass development of cutting-edge genetic therapies. The centre aims to develop therapies for many devastating genetic disorders that are currently untreatable, such as rare disorders that cause severe seizures in infants and neurodevelopmental delay, certain types of blindness and immune disorders, and severe neurological disorders such as Huntington’s Disease. Read more: https://lnkd.in/di_ThKsq
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Delighted to see further success today for AlveoGene, with its novel inhaled gene therapy receiving Orphan Drug Designation from the FDA for the rare disease Lethal Neonatal Surfactant Protein B (SP-B) Deficiency. AlveoGene was founded in 2023 as the first company being advanced under the Oxford-Harrington Rare Disease Centre Therapeutics Accelerator, with co-investment from Oxford Science Enterprises and Harrington Discovery Institute at University Hospitals This announcement follows the recent Paediatric Rare Diseases Designation by the FDA for AVG-002, which represents a potential first therapeutic intervention for this critical unmet medical need. Read more: https://lnkd.in/dinhGZZM
OHC Therapeutics Accelerator Company, AlveoGene, Receives Orphan Drug Designation from FDA for Novel Inhaled Gene Therapy
oxfordharrington.org
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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 here: https://lnkd.in/dQ_yUYAg
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A powerful reminder from Julia Vitarello of the daily lives of families affected by rare diseases. Raiden Pham is 4 years old and is diagnosed with a rare neurodegenerative condition, UBA5 disorder. As with thousands of children with rare genetic conditions, hope for Raiden currently lies in the hands of his parents Raiden Science Foundation. More here https://lnkd.in/gdncyKTk
Mila’s mom, founder/co-founder Mila's Miracle Foundation, N=1 Collaborative, EveryONE Medicines
To survive each day, Raiden's parents always need a plan. They don't have the luxury of just waking up and letting the day unfold. Every few hours they have to ask themselves, "Did we feed him?", "Did we give him water?", "Did we give him his medicines?". And even though they are juggling their jobs, their other child and putting dinner on the table, they can't rush with Raiden or he vomits and could aspirate. It's hard to imagine adding anything more to Raiden's life or to their lives, but they live in fear of the day that he begins having seizures, something he hasn't experienced yet, but is only a matter of time with his disease. And so they do the only thing they can... take each day at a time. This is Today: https://lnkd.in/dUUzDFB Photo Credit: Ulli Bonnekamp Linda Pham Tommy Pham ⚡️ Raiden Science Foundation
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Delighted to see Mila's Legacy receiving this award, recognising the thoughtful and impactful work of Sandra Kanthal and Natasha Loder exploring the transformative potential of individualised therapies for children with rare diseases. Featuring OHC Director Matthew Wood, listen here on BBC Radio 4 “Mila’s Legacy”: https://lnkd.in/guVQc_zH
Congratulations to Sandra Kanthal and Natasha Loder for winning this year’s Gold Award for the BBC Radio 4 audio documentary, “Mila’s Legacy,” presented by the prestigious and internationally-recognized AAAS Kavli Science Journalism Awards. I’m very grateful for the incredibly careful and thorough coverage of Mila’s story and the revolution of individualized medicine I am helping lead. “Some stories resonate long after they happen. And some change the world. Julia and Mila’s story felt like one of those, and it was an honor to tell it in the hope that it might help other children survive a deadly diagnosis.” - Natasha Loder & Sandra Kanthal BBC Radio 4 “Mila’s Legacy”: https://lnkd.in/guVQc_zH AAAS Kavli Science Journalism Awards: https://lnkd.in/g2sXiCdR Sandra Kanthal Natasha Loder BBC American Association for the Advancement of Science The Kavli Foundation Medicines and Healthcare products Regulatory Agency Genomics England Oxford-Harrington Rare Disease Centre University of Oxford Tim Yu
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Excellent news for AlveoGene, making progress on the regulatory path to a gene therapy for a rare, lethal neonatal deficiency. Read more: https://lnkd.in/dZnyrT5Z
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Great news for those who couldn't attend the Oxford-Harrington Rare Disease Symposium on September 24-26th! Video recordings of 16 symposium talks from experts in rare disease therapeutics development are now available online. Find the video highlights linked in the story below: https://lnkd.in/d2rgNBMX
Oxford-Harrington Rare Disease Centre Symposium 2024 Video Highlights
oxfordharrington.org