Contact information

PA
Miss Sahar Kiani
maclaren@eye.ox.ac.uk

Colleges

Merton College

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.

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Research Group

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

Clinical Trials
Principal Investigator
Fellow of Merton College
Bodley Fellow - Medical Sciences and Ophthalmology
Beacon Therapeutics Inc
Scientific Co-Founder
Euretina
Member of the Inherited Retinal Diseases Section

Long term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy

Hankins MW. et al, (2017), PNAS

Recent publications

Cataract surgery with autosomal recessive cornea plana caused by a novel KERA mutation.

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.

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.

Christou EE. et al, (2026), Graefes Arch Clin Exp Ophthalmol

Investigating ethnicity-related variability in the human L-cone spectral sensitivity function

MORIMOTO T. et al, (2026), Vision Research

DIRECT POSTERIOR RETINAL INJURY CAUSED BY INTRAVITREAL INJECTIONS: Management and Outcomes From a Vitreoretinal Perspective.

Christou EE. et al, (2026), Retina, 46, 272 - 280

Multimodal imaging and electrophysiological features in bradyopsia associated with homozygous variants (c.895T>C) in Regulator of G-protein Signaling 9 (RGS9).

Borchert GA. et al, (2026), Ophthalmic Genet, 47, 14 - 21

Genotype-Phenotype Correlations in PRPH2 Retinopathies: A Comprehensive Analysis of 36 Patients from the Oxford Eye Hospital, UK.

Al-Khuzaei S. et al, (2025), Genes (Basel), 16

Single-cell and spatial transcriptomic analyses of gene therapy-associated retinal inflammation in non-human primates

Sourd C. et al, (2025)

Exploring standard and low luminance visual acuity and the Moorfields Acuity Chart as outcome measures in inherited retinal disease.

Taylor LJ. et al, (2025), Ophthalmic Physiol Opt, 45, 1158 - 1163

Characterizing Inner Retinal Changes in End-Stage Inherited Retinal Diseases That Might be Suitable for Optogenetic Therapies.

Ng BWJ. et al, (2025), Transl Vis Sci Technol, 14

An Analysis of Scotopic Microperimetry in Healthy Adults.

Adeyoju DAO. et al, (2025), Transl Vis Sci Technol, 14

Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy.

Purdy R. et al, (2025), Prog Retin Eye Res, 106

Spontaneously Immortalised Nonhuman Primate Müller Glia Cell Lines as Source to Explore Retinal Reprogramming Mechanisms for Cell Therapies.

Salman A. et al, (2025), J Cell Physiol, 240

XOLARIS: A 24-Month, Prospective, Natural History Study of 201 Participants with Retinitis Pigmentosa GTPase Regulator-Associated X-Linked Retinitis Pigmentosa.

MacLaren RE. et al, (2025), Ophthalmol Sci, 5

Early-Onset Cone Photoreceptor Degeneration Is Associated With High Myopia in RPGR-Related Retinal Dystrophy.

Raji S. et al, (2025), J Ophthalmol, 2025

Retinal Characteristics of Female Choroideremia Carriers: Multimodal Imaging, Microperimetry, and Genetics.

Gocuk SA. et al, (2024), Ophthalmol Retina, 8, 1200 - 1210

Expanding the genotypic and phenotypic spectra with a novel variant in the ciliopathy gene, CFAP410, associated with selective cone degeneration.

Borchert GA. et al, (2024), Ophthalmic Genet, 45, 633 - 639

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.

Borchert GA. et al, (2024), Eye (Lond), 38, 3569 - 3577

Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study.

Lam BL. et al, (2024), Ophthalmology, 131, 1083 - 1093

Gene Therapies in Clinical Development to Treat Retinal Disorders.

McClements ME. et al, (2024), Mol Diagn Ther, 28, 575 - 591

Retinal Focal Nodular Gliosis (Vasoproliferative Tumors) Have Varied Clinical Courses Requiring Tailored Management: A Case Series.

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.

Quinn J. et al, (2024), Sci Rep, 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.

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.

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.

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.

Shamsnajafabadi H. et al, (2024), Genes (Basel), 15

Rescue of cone and rod photoreceptor function in a CDHR1-model of age-related retinal degeneration.

Yusuf IH. et al, (2024), Mol Ther, 32, 1445 - 1460

Gene therapy for choroideremia using an adeno-associated viral vector encoding Rab escort protein 1: the REGENERATE open-label trial

Cehajic-Kapetanovic J. et al, (2024), Efficacy and Mechanism Evaluation, 1 - 59

A hypomorphic variant of choroideremia is associated with a novel intronic mutation that leads to exon skipping.

Waldock WJ. et al, (2024), Ophthalmic Genet, 45, 210 - 217

Hypomorphic CDHR1 variants may result in retinitis pigmentosa with relative preservation of cone function.

Farag S. et al, (2024), Ophthalmic Genet, 45, 201 - 206

Randomised controlled trial of adjunctive triamcinolone acetonide in eyes undergoing vitreoretinal surgery following open globe trauma: The ASCOT study.

Casswell EJ. et al, (2024), Br J Ophthalmol, 108, 440 - 448

IMPG2-Related Maculopathy.

Birtel J. et al, (2024), Am J Ophthalmol, 258, 32 - 42

Optical Coherence Tomography Feature of Retinoschisis in CRB1-Associated Maculopathy.

Bellingrath J-S. et al, (2024), JAMA Ophthalmol, 142, 158 - 161

CRISPR Manipulation of Age-Related Macular Degeneration Haplotypes in the Complement System: Potential Future Therapeutic Applications/Avenues.

Salman A. et al, (2024), Int J Mol Sci, 25

Age-related macular degeneration: suitability of optogenetic therapy for geographic atrophy.

Borchert GA. et al, (2024), Front Neurosci, 18

Is RPGR-related retinal dystrophy associated with systemic disease? A case series.

Han RC. et al, (2023), Ophthalmic Genet, 44, 577 - 584

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.

Fry LE. and MacLaren RE., (2023), Genes (Basel), 14

Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center.

Kiraly P. et al, (2023), Biomolecules, 13

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