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
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
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
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia
Journal article
Xue K. et al, (2018), Nature Medicine, 24, 1507 - 1512
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
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
Transplanted photoreceptor precursors transfer proteins to host photoreceptors by a mechanism of cytoplasmic fusion
Journal article
Singh MS. et al, (2016), Nature Communications, 7
Visual Acuity after Retinal Gene Therapy for Choroideremia
Journal article
Edwards TL. et al, (2016), New England Journal of Medicine, 374, 1996 - 1998
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
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
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
Retinal repair by transplantation of photoreceptor precursors
Journal article
MacLaren RE. et al, (2006), Nature, 444, 203 - 207
Recent publications
Investigating ethnicity-related variability in the human L-cone spectral sensitivity function
Journal article
Schneider AC. et al, (2026), Vision Research, 243, 108769 - 108769
Cataract surgery with autosomal recessive cornea plana caused by a novel KERA mutation.
Journal article
Abdalla Elsayed MEA. and MacLaren RE., (2026), Am J Ophthalmol Case Rep, 41
Geographic atrophy in age-related macular degeneration: phenotypic characterisation for clinical trial consideration.
Journal article
Borchert GA. et al, (2026), Graefes Arch Clin Exp Ophthalmol
A literature review of macular holes in age-related macular degeneration: insights into the pathogenesis, prognosis and surgical outcomes.
Journal article
Christou EE. et al, (2026), Graefes Arch Clin Exp Ophthalmol
Central Retinal Sensitivity Decline in RPGR-Related Retinal Phenotypes
Journal article
Josan AS. et al, (2026), American Journal of Ophthalmology, 282, 154 - 161
Multimodal imaging and electrophysiological features in bradyopsia associated with homozygous variants (c.895T>C) in Regulator of G-protein Signaling 9 (RGS9).
Journal article
Borchert GA. et al, (2026), Ophthalmic Genet, 47, 14 - 21
DIRECT POSTERIOR RETINAL INJURY CAUSED BY INTRAVITREAL INJECTIONS: Management and Outcomes From a Vitreoretinal Perspective.
Journal article
Christou EE. et al, (2026), Retina, 46, 272 - 280
A novel EYS c.6192-1G>A variant presents ideal base editing therapeutic opportunities.
Journal article
Kaukonen M. et al, (2026), Ophthalmic Genet, 1 - 4
ABCA4-associated maculopathy suspected to be ocular toxoplasmosis.
Journal article
Elsayed MEAA. et al, (2026), BMC Ophthalmol, 26
Low-luminance visual acuity and low-luminance deficit: optimising measurement and analysis
Journal article
Forte G. et al, (2026), Clinical and Experimental Optometry, 109, 53 - 59
IMPG2-associated retinal dystrophy with a novel missense variant and therapeutic options via adenine base editing
Journal article
Abdalla Elsayed MEA. et al, (2026), Ophthalmic Genetics, 1 - 7
Expanding the Genetic Spectrum in IMPG1 and IMPG2 Retinopathy
Journal article
Al-Khuzaei S. et al, (2025), Genes, 16, 1474 - 1474
Gene Therapy Strategies for the Treatment of Bestrophinopathies.
Journal article
Haldrup SB. et al, (2025), Int J Mol Sci, 26
Genotype–Phenotype Correlations in PRPH2 Retinopathies: A Comprehensive Analysis of 36 Patients from the Oxford Eye Hospital, UK
Journal article
Al-Khuzaei S. et al, (2025), Genes, 16, 1016 - 1016
Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates
Preprint
Sourd C. et al, (2025)
Exploring standard and low luminance visual acuity and the Moorfields Acuity Chart as outcome measures in inherited retinal disease
Journal article
Taylor LJ. et al, (2025), Ophthalmic and Physiological Optics, 45, 1158 - 1163
An Analysis of Scotopic Microperimetry in Healthy Adults
Journal article
Adeyoju DAO. et al, (2025), Translational Vision Science & Technology, 14, 18 - 18
Characterizing Inner Retinal Changes in End-Stage Inherited Retinal Diseases That Might be Suitable for Optogenetic Therapies.
Journal article
Ng BWJ. et al, (2025), Transl Vis Sci Technol, 14
Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy
Journal article
Purdy R. et al, (2025), Progress in Retinal and Eye Research, 106, 101354 - 101354
CRISPR targeting of SNPs associated with age-related macular degeneration in ARPE-19 cells: a potential model for manipulating the complement system
Journal article
Salman A. et al, (2025), Gene Therapy, 32, 132 - 141
Gene therapy for choroideremia: progress, potential and pitfalls.
Journal article
Abdalla Elsayed MEA. et al, (2025), Expert Opin Biol Ther, 25, 257 - 263
Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration
Journal article
Fry LE. et al, (2025), Communications Biology, 8
Longitudinal assessment of female carriers of choroideremia using multimodal retinal imaging
Journal article
Gocuk SA. et al, (2025), British Journal of Ophthalmology, 109, 293 - 299
Spontaneously Immortalised Nonhuman Primate Müller Glia Cell Lines as Source to Explore Retinal Reprogramming Mechanisms for Cell Therapies
Journal article
Salman A. et al, (2025), Journal of Cellular Physiology, 240
Early‐Onset Cone Photoreceptor Degeneration Is Associated With High Myopia in RPGR ‐Related Retinal Dystrophy
Conference paper
Raji S. et al, (2025), Journal of Ophthalmology, 2025
XOLARIS: A 24-Month, Prospective, Natural History Study of 201 Participants with Retinitis Pigmentosa GTPase Regulator-Associated X-Linked Retinitis Pigmentosa
Journal article
MacLaren RE. et al, (2025), Ophthalmology Science, 5, 100595 - 100595
Retinal Characteristics of Female Choroideremia Carriers
Journal article
Gocuk SA. et al, (2024), Ophthalmology Retina, 8, 1200 - 1210
Real-world six-month outcomes in patients switched to faricimab following partial response to anti-VEGF therapy for neovascular age-related macular degeneration and diabetic macular oedema.
Journal article
Borchert GA. et al, (2024), Eye (Lond), 38, 3569 - 3577
Expanding the genotypic and phenotypic spectra with a novel variant in the ciliopathy gene, CFAP410, associated with selective cone degeneration.
Journal article
Borchert GA. et al, (2024), Ophthalmic Genet, 45, 633 - 639
Retinal Patterns and the Role of Autofluorescence in Choroideremia.
Journal article
Poli FE. et al, (2024), Genes (Basel), 15
A novel frameshift variant in LAMP2 gene mimicking choroideremia carrier retinopathy
Journal article
Narayan A. et al, (2024), Ophthalmic Genetics, 45, 668 - 675
Exploring Scotopic Microperimetry as an Outcome Measure in Choroideremia
Journal article
Taylor LJ. et al, (2024), Translational Vision Science & Technology, 13, 29 - 29
Establishing Clinical Trial Endpoints in Selecting Patients for RPGR Retinal Gene Therapy
Journal article
Christou EE. et al, (2024), Translational Vision Science & Technology, 13, 18 - 18
Gene therapy in the early stages of retinal degeneration.
Journal article
MacLaren RE., (2024), Lancet, 404, 911 - 913
Investigating the impact of asymmetric macular sensitivity on visual acuity chart reading in choroideremia
Journal article
Baffour‐Awuah KA. et al, (2024), Ophthalmic and Physiological Optics, 44, 1188 - 1201
Gene Therapies in Clinical Development to Treat Retinal Disorders
Journal article
McClements ME. et al, (2024), Molecular Diagnosis & Therapy, 28, 575 - 591
Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study
Journal article
Lam BL. et al, (2024), Ophthalmology, 131, 1083 - 1093
Genetic therapies and potential therapeutic applications of CRISPR activators in the eye.
Journal article
Ng BW. et al, (2024), Prog Retin Eye Res, 102
Retinal Focal Nodular Gliosis (Vasoproliferative Tumors) Have Varied Clinical Courses Requiring Tailored Management: A Case Series.
Journal article
Ie A. et al, (2024), Ocul Oncol Pathol, 10, 175 - 181
Single-cell transcriptomic analysis of retinal immune regulation and blood-retinal barrier function during experimental autoimmune uveitis
Journal article
Quinn J. et al, (2024), Scientific Reports, 14
An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study.
Journal article
MacLaren RE. et al, (2024), Hum Gene Ther, 35, 564 - 575
A Prospective, Observational, Non-interventional Clinical Study of Participants With Choroideremia: The NIGHT Study.
Journal article
Maclaren RE. et al, (2024), Am J Ophthalmol, 263, 35 - 49
Robot-Assisted Eye Surgery: A Systematic Review of Effectiveness, Safety, and Practicality in Clinical Settings.
Journal article
Thirunavukarasu AJ. et al, (2024), Transl Vis Sci Technol, 13
In Silico CRISPR-Cas-Mediated Base Editing Strategies for Early-Onset, Severe Cone-Rod Retinal Degeneration in Three Crumbs homolog 1 Patients, including the Novel Variant c.2833G>A.
Journal article
Shamsnajafabadi H. et al, (2024), Genes (Basel), 15
Gene therapy for choroideremia using an adeno-associated viral vector encoding Rab escort protein 1: the REGENERATE open-label trial
Journal article
Cehajic-Kapetanovic J. et al, (2024), Efficacy and Mechanism Evaluation, 1 - 59
Rescue of cone and rod photoreceptor function in a CDHR1-model of age-related retinal degeneration
Journal article
Yusuf IH. et al, (2024), Molecular Therapy, 32, 1445 - 1460
A hypomorphic variant of choroideremia is associated with a novel intronic mutation that leads to exon skipping
Journal article
Waldock WJ. et al, (2024), Ophthalmic Genetics, 45, 210 - 217
Hypomorphic CDHR1 variants may result in retinitis pigmentosa with relative preservation of cone function
Journal article
Farag S. et al, (2024), Ophthalmic Genetics, 45, 201 - 206
Randomised controlled trial of adjunctive triamcinolone acetonide in eyes undergoing vitreoretinal surgery following open globe trauma: The ASCOT study.
Journal article
Casswell EJ. et al, (2024), Br J Ophthalmol, 108, 440 - 448
Optical Coherence Tomography Feature of Retinoschisis in CRB1-Associated Maculopathy
Journal article
Bellingrath J-S. et al, (2024), JAMA Ophthalmology, 142, 158 - 158
CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues
Journal article
Salman A. et al, (2024), International Journal of Molecular Sciences, 25, 1697 - 1697
Age-related macular degeneration: suitability of optogenetic therapy for geographic atrophy.
Journal article
Borchert GA. et al, (2024), Front Neurosci, 18
Choroideremia: Toward Regulatory Approval of Retinal Gene Therapy
Journal article
Yusuf IH. and MacLaren RE., (2023), Cold Spring Harbor Perspectives in Medicine, 13, a041279 - a041279
Comment on Di Giosaffatte et al. A Novel Hypothesis on Choroideremia-Manifesting Female Carriers: Could CHM In-Frame Variants Exert a Dominant Negative Effect? A Case Report. Genes 2022, 13, 1268.
Journal article
Fry LE. and MacLaren RE., (2023), Genes (Basel), 14
Is RPGR-related retinal dystrophy associated with systemic disease? A case series
Journal article
Han RC. et al, (2023), Ophthalmic Genetics, 44, 577 - 584
Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center
Journal article
Kiraly P. et al, (2023), Biomolecules, 13, 1484 - 1484
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
Correlation Between Fundus Autofluorescence Pattern and Retinal Function on Microperimetry in Choroideremia
Journal article
Poli FE. et al, (2023), Translational Vision Science & Technology, 12, 24 - 24