About

Peter Anthony Campochiaro, M.D., is the George S. and Dolores Doré Eccles Professor of Ophthalmology and Neuroscience at The Wilmer Eye Institute, Johns Hopkins University School of Medicine. Born on September 23, 1952, in Cohoes, New York, Dr. Campochiaro completed his B.S. at the University of Notre Dame, graduating magna cum laude and Phi Beta Kappa, followed by his M.D. at Johns Hopkins University School of Medicine. His academic career includes appointments as Assistant, Associate, and full Professor of Ophthalmology at the University of Virginia School of Medicine before joining Johns Hopkins, where he has served as Professor of Ophthalmology and Neuroscience and Director of Vitreoretinal Surgery. Dr. Campochiaro's research focuses on retinal diseases, particularly mechanisms underlying ocular neovascularization, diabetic retinopathy, macular edema, and retinal degenerations such as retinitis pigmentosa. He has contributed extensively to understanding the roles of growth factors like PDGFs and FGFs, oxidative damage, and molecular pathways involved in retinal pathology. His work includes pioneering studies on gene therapy, anti-angiogenic agents, and novel peptide inhibitors for ocular diseases. Dr. Campochiaro has received numerous honors, including the Rosenthal Award from the Macula Society, the Proctor Medal from the Association for Research in Vision and Ophthalmology, and election to the National Academy of Inventors. He has been continuously funded by the NIH and other agencies for research on suprachoroidal gene transfer, peptide therapeutics, and biomaterial inhibitors targeting retinal and choroidal vascular diseases. His professional affiliations include the American Academy of Ophthalmology, the Association for Research in Vision and Ophthalmology, and the Retina Society.

Research topics

• Medicine
• Ophthalmology
• Internal medicine
• Pharmacology
• Biology
• Endocrinology
• Genetics
• Cancer research
• Pathology
• Composite material
• Neuroscience
• Nanotechnology
• Surgery
• Biomedical engineering
• Cardiology
• Computational biology
• Materials science
• Chemistry
• Chromatography

Selected publications

• Rabbit model of oxidative stress-induced retinal degeneration

  Free Radical Biology and Medicine · 2025-02-20 · 2 citations

  articleOpen accessSenior author
  PublisherOA PDFDOI

• Gene therapy for AMD: better as an adjuvant than a replacement – Authors' reply

  The Lancet · 2025-03-01

  letter1st authorCorresponding
  PublisherDOI

• Stair-Case/Honeycomb Maculopathy in Alport Syndrome: A Case Report

  Case Reports in Ophthalmology · 2025-06-24

  articleOpen accessSenior author

  Introduction: Alport syndrome is an inherited disease caused by mutations in COL4A5, COLA3, or COL4A4 resulting in kidney failure, hearing loss, and ocular symptoms. We report a patient with Alport syndrome who has a "stair-case/honeycomb" maculopathy, a rare but distinctive finding in this disease. Case Presentation: A 53-year-old man with Alport syndrome was referred for gradual decrease in vision. His ocular history was remarkable for intraocular lens implantation secondary to lenticonus in each eye. Fundus photography showed rare white dots in the temporal mid-periphery in each eye and fundus autofluorescence was normal. Optical coherence tomography (OCT) B-scans through the fovea showed irregular thinning of the inner retina with peaks and valleys in the macula of each eye. The ellipsoid zone was intact except for mild patchiness centrally. En face retinal structural OCT angiography (OCTA) images showed a mosaic-like honeycomb pattern in the macular region in both eyes, with hyporeflective depressions in areas of focal retinal atrophy. Retinal OCTA scans showed irregular foveal avascular zone (FAZ) areas with capillaries crossing the FAZ in the left eye, corresponding to islands of preserved retinal tissue. There was predominance of capillaries in the deeper retinal layers centrally. Conclusion: While severe irregular thinning of the macula is not a common feature in Alport syndrome, when it is present in patients who have not been previously diagnosed, particularly in patients with renal disease, it should suggest the diagnosis of Alport syndrome. Its occurrence can be the cause of vision loss which is not commonly associated with Alport central maculopathy.

  PublisherOA PDFDOI

• Bispecific receptor decoy proteins block ocular neovascularization via simultaneous blockade of vascular endothelial growth factor A and C

  Molecular Therapy · 2025-03-26 · 2 citations

  article
  PublisherDOI

• Continuous Ranibizumab via Port Delivery System vs Monthly Ranibizumab for Treatment of Diabetic Macular Edema

  JAMA Ophthalmology · 2025-03-06 · 16 citations

  letterOpen access

  Importance: Frequent visits and intravitreal anti-vascular endothelial growth factor (VEGF) injections are often required to manage diabetic macular edema (DME), burdening patients and their health care networks. The Port Delivery System (PDS) with ranibizumab is the first continuous anti-VEGF therapy that has the potential to reduce visit and treatment burden without sacrificing vision outcomes for patients with DME. Objective: To evaluate the efficacy and safety through 64 weeks of ranibizumab, 100 mg/mL, via PDS with refill exchanges every 24 weeks (PDS Q24W) in patients with DME vs intravitreal injections of ranibizumab, 0.5 mg, every 4 weeks (monthly ranibizumab). Design, Setting, and Participants: This randomized clinical trial was a phase 3, multicenter, noninferiority trial conducted across 87 sites in the US. Treatment-naïve patients at least 18 years old with center-involved DME were eligible for study participation. Enrollment was from September 30, 2019, to June 25, 2021; data were analyzed from September 30, 2019, to September 19, 2022. Intervention: Participants were randomized 3:2 to receive 4 monthly doses of ranibizumab, 0.5 mg, followed by ranibizumab, 100 mg/mL, via PDS Q24W or monthly ranibizumab. Main Outcome and Measure: The primary end point was change in best-corrected visual acuity (BCVA) from baseline averaged over weeks 60 and 64. Results: A total of 634 participants were randomized (PDS Q24W group, n = 381; monthly ranibizumab, n = 253). The mean (SD) age at baseline was 60.7 (9.6) years; 363 (57.3%) participants were male and 271 (42.7%) female. Adjusted mean BCVA change from baseline averaged over weeks 60 and 64 was an increase of 9.6 letters for PDS Q24W and 9.4 letters for monthly ranibizumab (difference, 0.2; 95% CI, -1.2 to 1.6), meeting the primary end point of PDS noninferiority (margin, -4.5 letters). PDS Q24W participants had a mean (SD) decrease of 6.7 (12.0) letters 4 weeks after PDS insertion; mean BCVA was similar to monthly ranibizumab 16 weeks after implantation. Adverse events of special interest were more common in the PDS Q24W group (88 participants; 27.5%) than the monthly ranibizumab group (28 participants; 8.9%). No cases of endophthalmitis or retinal detachment were reported with PDS Q24W. Conclusions and Relevance: This trial found that changes in BCVA from baseline averaged over weeks 60/64 in the PDS Q24W group were comparable to the monthly ranibizumab group. While AESIs were more common with PDS Q24W, there were no instances of endophthalmitis or retinal detachment. Continuous ranibizumab, 100 mg/mL, via PDS was approved in the US for patients with DME in February 2025 and provides effective, durable, and generally well-tolerated treatment for DME with retreatment every 6 months through at least 64 weeks. Trial Registration: ClinicalTrials.gov Identifier: NCT04108156.

  PublisherOA PDFDOI

• Suprachoroidal gene transfer with nonviral nanoparticles in large animal eyes

  Science Advances · 2024-03-08 · 19 citations

  articleOpen accessSenior authorCorresponding

  Suprachoroidal nonviral gene therapy with biodegradable poly(β-amino ester) nanoparticles (NPs) provides widespread expression in photoreceptors and retinal pigmented epithelial (RPE) cells and therapeutic benefits in rodents. Here, we show in a human-sized minipig eye that suprachoroidal injection of 50 μl of NPs containing 19.2 μg of GFP expression plasmid caused GFP expression in photoreceptors and RPE throughout the entire eye with no toxicity. Two weeks after injection of 50, 100, or 200 μl, there was considerable within-eye and between-eye variability in expression that was reduced 3 months after injection of 200 μl and markedly reduced after three suprachoroidal injections at different locations around the eye. Reduction of bacterial CpG sequences in the expression plasmid resulted in a trend toward higher expression. These data indicate that nonviral suprachoroidal gene therapy with optimized polymer, expression plasmid, and injection approach has potential for treating photoreceptors throughout the entire retina of a human-sized eye.

  PublisherDOI

• TENAYA and LUCERNE

  Ophthalmology · 2024 · 163 citations

  • Medicine

  PURPOSE: To evaluate 2-year efficacy, durability, and safety of the bispecific antibody faricimab, which inhibits both angiopoietin-2 and VEGF-A. DESIGN: TENAYA (ClinicalTrials.gov identifier, NCT03823287) and LUCERNE (ClinicalTrials.gov identifier, NCT03823300) were identically designed, randomized, double-masked, active comparator-controlled phase 3 noninferiority trials. PARTICIPANTS: Treatment-naive patients with neovascular age-related macular degeneration (nAMD) 50 years of age or older. METHODS: Patients were randomized (1:1) to intravitreal faricimab 6.0 mg up to every 16 weeks (Q16W) or aflibercept 2.0 mg every 8 weeks (Q8W). Faricimab fixed dosing based on protocol-defined disease activity at weeks 20 and 24 up to week 60, followed up to week 108 by a treat-and-extend personalized treatment interval regimen. MAIN OUTCOME MEASURES: Efficacy analyses included change in best-corrected visual acuity (BCVA) from baseline at 2 years (averaged over weeks 104, 108, and 112) and proportion of patients receiving Q16W, every 12 weeks (Q12W), and Q8W dosing at week 112 in the intention-to-treat population. Safety analyses included ocular adverse events (AEs) in the study eye through study end at week 112. RESULTS: Of 1326 patients treated across TENAYA/LUCERNE, 1113 (83.9%) completed treatment (n = 555 faricimab; n = 558 aflibercept). The BCVA change from baseline at 2 years was comparable between faricimab and aflibercept groups in TENAYA (adjusted mean change, +3.7 letters [95% confidence interval (CI), +2.1 to +5.4] and +3.3 letters [95% CI, +1.7 to +4.9], respectively; mean difference, +0.4 letters [95% CI, -1.9 to +2.8]) and LUCERNE (adjusted mean change, +5.0 letters [95% CI, +3.4 to +6.6] and +5.2 letters [95% CI, +3.6 to +6.8], respectively; mean difference, -0.2 letters [95% CI, -2.4 to +2.1]). At week 112 in TENAYA and LUCERNE, 59.0% and 66.9%, respectively, achieved Q16W faricimab dosing, increasing from year 1, and 74.1% and 81.2%, achieved Q12W or longer dosing. Ocular AEs in the study eye were comparable between faricimab and aflibercept groups in TENAYA (55.0% and 56.5% of patients, respectively) and LUCERNE (52.9% and 47.5% of patients, respectively) through week 112. CONCLUSIONS: Treat-and-extend faricimab treatment based on nAMD disease activity maintained vision gains through year 2, with most patients achieving extended dosing intervals. FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.

  PublisherOA PDFDOI

• Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study

  The Lancet · 2024-03-27 · 110 citations

  article1st authorCorresponding
  PublisherDOI

• Rabbit Model of Oxidative Stress-Induced Retinal Degeneration

  SSRN Electronic Journal · 2024-01-01

  preprintOpen accessSenior author
  PublisherDOI

• Heterogeneity in disease activity, frequency of treatments, and visual outcomes among patients with retinal vein occlusion: relationship between injection need and vision with as-needed ranibizumab

  British Journal of Ophthalmology · 2024-01-30 · 5 citations

  articleOpen access

  BACKGROUND/AIMS: We characterised the relationships between monitoring frequency, ranibizumab injection need and vision in patients receiving as-needed (pro re nata; PRN) ranibizumab for macular oedema due to branch retinal vein occlusion (BRVO) or central retinal vein occlusion (CRVO) in this post-hoc analysis of SHORE and HORIZON. METHODS: Patients aged 18 years and older with macular oedema due to BRVO/CRVO were included in this analysis. Injection frequency and best-corrected visual acuity (BCVA) were evaluated by PRN injection frequency in the PRN dosing phase (months (M) 7-15) of SHORE and through 12 months of HORIZON. Prespecified PRN re-treatment criteria for each trial were based on protocol-prespecified BCVA and optical coherence tomography outcomes. RESULTS: After the initial 7 monthly ranibizumab injections, patients in SHORE gained a mean of 18.3 letters from baseline. Patients randomised to PRN, on average, maintained these gains. However, some patients experienced additional mean gains, whereas others suffered losses (range 4.0 (95% CI 0.7 to 7.3) to -4.6 (95% CI -11.8 to 2.6) letters in patients who received 0 and 6-7 PRN injections, respectively). In BRAVO and CRUISE (lead-in trials), patients experienced mean gains from baseline to M6 (monthly dosing) of 19.3 and 15.0 letters, respectively, with gains maintained with PRN from M6 to M12. However, mean BCVA changes from baseline to M12 varied in HORIZON (range -0.4 (95% CI -2.5 to 1.6) to -3.6 (95% CI -6.2 to -1.0) letters in patients who received zero and six injections, respectively, during the preceding PRN phase of BRAVO and CRUISE). CONCLUSION: The BRVO/CRVO population is heterogenous with a varied response to ranibizumab treatment.

  PublisherOA PDFDOI

Recent grants

• Suprachoroidal nonviral gene transfer of engineered VEGF antagonists

  NIH · $2.4M · 2020–2025

• A novel hypotonic gelling eye drop for topical treatment of retinal degenerative diseases

  NIH · $1.6M · 2020–2023

• Integrin-binding Peptide for Ocular Neovascularization and Macular Edema: Molecular Mechanism of Action

  NIH · $2.3M · 2019–2028

• New Treatments of Ocular Neovascularization and Macular Edema

  NIH · $5.5M · 1999–2016

• Sustained Suprachoroidal Delivery of Therapeutic Peptidesfor Ocular Diseases

  NIH · $449k · 2016–2019

Frequent coauthors

• Jikui Shen

  Johns Hopkins Medicine

  219 shared

• Sean F. Hackett

  Nottingham University Hospitals NHS Trust

  193 shared

• Donald J. Zack

  Johns Hopkins University

  178 shared

• Sean F. Hackett

  Johns Hopkins Medicine

  159 shared

• Stanley A. Vinores

  Johns Hopkins University

  117 shared

• Syed Mahmood Shah

  Sher-e-Kashmir University of Agricultural Sciences and Technology of Kashmir

  112 shared

• Yogita Kanan

  95 shared

• Mahmood Khan

  93 shared

Labs

• Peter Campochiaro's LabPI

  The provided HTML does not contain a description of the lab's research focus.