Colleges
Biography
Robert MacLaren is Professor of Ophthalmology at the University of Oxford, Consultant Ophthalmologist at the Oxford Eye Hospital, Honorary Professor of Ophthalmology at the UCL Institute of Ophthalmology, Honorary Consultant Vitreoretinal Surgeon at Moorfields Eye Hospital and an NIHR Senior Investigator. He is also a Fellow of the Royal College of Ophthalmologists, Fellow and former King James IV Professor of Surgery at the Royal College of Surgeons of Edinburgh, Fellow of the American College of Surgeons, Fellow of the Academy of Medical Sciences, Bodley Fellow of Merton College Oxford and Civilian Consultant Advisor to the Royal Navy. He has been a recipient of the ARVO Camras Award for Translational Research, the RP Fighting Blindness Scientist of the Year Award, the American Academy of Ophthalmology Achievement Award, the Royal College of Ophthalmologists Keeler Medal and the Macular Society Clinical Service of the Year award.
Together with the University of Oxford in 2014, he co-founded Nightstar Therapeutics in order to expand retinal gene therapy towards worldwide approval for patients with retinal diseases. After listing on NASDAQ in 2017, Nightstar Therapeutics was acquired by Biogen in 2019, making it one of the most successful British biotechnology company buyouts of all time. In 2023, he co-founded Beacon Therapeutics together with Syncona Partners, achieving the largest equity at launch for any Oxford spinout company to date. Over the last decade, he has helped raise over a billion dollars of funding for retinal gene therapy.
Robert MacLaren
MB ChB DPhil DSc FRCOphth FRCS FACS FMedSci
Professor of Ophthalmology
- Professor of Ophthalmology, University of Oxford
- Consultant Vitreoretinal Surgeon
- Fellow of Merton College
Research Summary
Our clinical and laboratory research is dedicated to finding new treatments for blindness, particularly in patients with incurable retinal diseases, using stem cell-based approaches, gene therapy or electronic retinas. We are also developing new techniques for cataract and retinal surgery. Our recent research achievements include:
- 2021: First demonstration of RNA silencing in vivo using self-cleaving mirtons applied to treat a dominantly inherited retinal degeneration (Nat Commun, 2021)
- 2020: Highest visual acuity ever recorded in a blind patient using a surgically implanted bionic eye, in collaboration with Retina Implant AG (Acta Ophthalmol, 2020)
- 2020: First gene therapy treatment available through the NHS following the NICE approval of Luxturna as a treatment for childhood blindness (BBC News).
- 2019: First in human gene therapy trial targeting the genetic mechanism of age-related macular degeneration, in collaboration with Gyroscope Therapeutics (BBC News).
- 2018: First in human gene therapy trial for choroideremia, meeting the primary endpoint of showing beneficial effects on vision (Nature Medicine, 2018).
- 2017: First in human gene therapy clinical trial for X-linked retinitis pigmentosa and showing efficacy with the vector developed in Oxford (Mol Ther, 2017), in collaboration with Nightstar Therapeutics (Nature Medicine, 2020).
- 2016: First in human use of a robot to operate inside the eye, in collaboration with Preceyes BV (Nature Biomedical Engineering, 2018).
- 2016: Long term efficacy of retinal gene therapy in choroideremia, leading to sustained improvement in visual acuity in patients over several years (New Eng J Med, 2016)
- 2015: Demonstration of efficacy of a new molecular treatment to prevent vitamin A dimerization in a mouse model of Stargardt disease (Proc Natl Acad Sci USA, 2015)
- 2014: Initial first in human clinical trial of gene therapy to treat choroideremia showing safety and early signs of efficacy (Lancet, 2014)
- 2013: Reconstruction of the outer retina using a cell therapy approach in mice that are completely blind from end-stage retinal degeneration (Proc Natl Acad Sci USA, 2013)
Websites
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Clinical Trials
Principal Investigator
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Fellow of Merton College
Bodley Fellow - Medical Sciences and Ophthalmology
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Beacon Therapeutics Inc
Scientific Co-Founder
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Euretina
Member of the Inherited Retinal Diseases Section
Key publications
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Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial
Journal article
MacLaren RE. et al, (2023), Nature Medicine, 29, 2464 - 2472
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Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR
Journal article
Cehajic-Kapetanovic J. et al, (2020), Nature Medicine, 26, 354 - 359
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Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia
Journal article
Xue K. et al, (2018), Nature Medicine, 24, 1507 - 1512
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First-in-human study of the safety and viability of intraocular robotic surgery
Journal article
Edwards TL. et al, (2018), Nature Biomedical Engineering, 2, 649 - 656
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Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy
Journal article
De Silva SR. et al, (2017), Proceedings of the National Academy of Sciences, 114, 11211 - 11216
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Visual Acuity after Retinal Gene Therapy for Choroideremia
Journal article
Edwards TL. et al, (2016), New England Journal of Medicine, 374, 1996 - 1998
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Transplanted photoreceptor precursors transfer proteins to host photoreceptors by a mechanism of cytoplasmic fusion
Journal article
Singh MS. et al, (2016), Nature Communications, 7
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Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization
Journal article
Charbel Issa P. et al, (2015), Proceedings of the National Academy of Sciences, 112, 8415 - 8420
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Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
Journal article
MacLaren RE. et al, (2014), The Lancet, 383, 1129 - 1137
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Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation
Journal article
Singh MS. et al, (2013), Proceedings of the National Academy of Sciences, 110, 1101 - 1106
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Retinal repair by transplantation of photoreceptor precursors
Journal article
MacLaren RE. et al, (2006), Nature, 444, 203 - 207