DelveInsight’s “Gene Therapy in Ophthalmology Market Report” provides a comprehensive overview of the therapeutic landscape, epidemiological trends, and market dynamics for gene therapies in key ophthalmic indications such as Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP), Choroideremia, and Age-related Macular Degeneration (AMD). The report covers major markets, including the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The ophthalmic gene therapy market is expected to witness significant growth through 2034, driven by advancements in vector delivery systems, increasing investment in R&D, and successful regulatory approvals like Luxturna (voretigene neparvovec-rzyl). A growing number of clinical trials, promising pipeline candidates, and supportive regulatory pathways are accelerating innovation across the sector.
With detailed analysis of key players, market drivers, barriers, competitive landscape, and emerging opportunities, this report serves as a vital resource for biopharma companies, investors, clinicians, and researchers seeking to understand the rapidly evolving gene therapy space in ophthalmology.
Some of the Key Facts of the Gene Therapy in Ophthalmology Market Report:
• According to DelveInsight, the U.S. led the 7MM Gene Therapy in Ophthalmology market in 2020, valued at around USD 35 million, with continued growth expected through 2034.
• LUXTURNA remains the only approved ophthalmic gene therapy, approved by the FDA in December 2017 and in the EU in November 2018.
• In 2023, the U.S. had an estimated 74,000 eligible Retinitis Pigmentosa cases for gene therapy.
• Estimated diagnosed prevalent cases in the U.S. (2023):
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Retinitis Pigmentosa: ~113,000
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Leber Congenital Amaurosis: ~10,000
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Achromatopsia: ~8,500
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Wet Age-Related Macular Degeneration (Wet-AMD): ~1.3 million
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Diabetic Retinopathy: ~5.1 million
• In May 2025, the FDA granted RMAT designation to Opus Genetics’ OPGx-LCA5 for treating Leber congenital amaurosis caused by LCA5 gene mutations. The AAV8-based gene therapy delivers a functional LCA5 gene to the outer retina. The designation was based on early data from an ongoing Phase I/II dose-escalation trial.
• In December 2024, eyeDNA Therapeutics received FDA Rare Pediatric Disease Designation for HORA-PDE6b, its gene therapy targeting inherited retinal dystrophy caused by PDE6b mutations.
• In December 2024, the FDA approved an investigational new drug application for SB-007, a gene therapy using an adeno-associated viral vector to target the genetic cause of Stargardt disease, as announced by SpliceBio.
• Leading companies in the gene therapy in ophthalmology market include Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, GenSight Biologics, Neuropht Therapeutics, Adverum Biotechnologies, EyeVensys, 4D Molecular Therapeutics, Exegenesis Bio, MeiraGTx, Johnson & Johnson Innovative Medicine, Neuropht Therapeutics, 4D Molecular Therapeutics, Atsena Therapeutics, Ocugen, AbbVie, REGENXBIO, Skyline Therapeutics, Huida Gene Therapeutics, Opus Genetics, and others.
• Emerging acute gene therapy in ophthalmology drugs include BOTARETIGENE SPAROPARVOVEC, AGTC-501, MCO-010, GS030, 4D 125, CTX PDE6B, OCU400, BS01, LUMEVOQ, NR082 (NFS-01), ADVM-022 (AAV.7M8-AFLIBERCEPT), JNJ-1887 (AAVCAGSCD59/JNJ-81201887), EYS 606, 4D 150, EXG102-031, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, NFS-02, 4D 110, ATSN-101, ATSN-201, OCU410, OCU410ST, AGTC-402, AGTC-401, ABBV-RGX-314, SKG0106, HG 004, OPGX-001, and others.
• Gene therapy is rapidly advancing in ophthalmology, driving demand for more effective treatments for inherited and hard-to-treat eye diseases.
To know in detail about the gene therapy in ophthalmology market outlook, drug uptake, treatment scenario, and epidemiology trends, click here: Gene Therapy in Ophthalmology Market Forecast
Gene Therapy in Ophthalmology Overview
Gene therapy is emerging as a transformative approach in ophthalmology, offering the potential to treat or even cure inherited retinal diseases (IRDs) and other ocular disorders at the genetic level. By delivering functional genes to replace or repair mutated ones, gene therapy addresses the root cause of vision loss rather than merely managing symptoms. The eye, with its immune-privileged status, small size, and ease of direct access, makes it an ideal organ for gene therapy interventions. Conditions such as Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP), X-linked Retinoschisis (XLRS), and age-related macular degeneration (AMD) are prime targets, with several therapies progressing through clinical trials.
The approval of Luxturna (voretigene neparvovec) by Spark Therapeutics for RPE65-mediated retinal dystrophy marked a milestone, proving the feasibility of gene therapy in ocular indications. Since then, the pipeline has expanded significantly with numerous biotech and pharmaceutical companies investing in adeno-associated virus (AAV) vectors, CRISPR-based editing, and non-viral delivery platforms. As understanding of disease genetics deepens and regulatory pathways become clearer, gene therapy in ophthalmology is poised to reshape the treatment landscape, offering hope for previously untreatable vision disorders.
Get a free sample of the gene therapy in ophthalmology market report with key insights and emerging therapies here: https://www.delveinsight.com/report-store/gene-therapy-in-ophthalmology-market
Gene Therapy in Ophthalmology Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Gene Therapy in Ophthalmology Epidemiology Segmentation:
The Gene Therapy in Ophthalmology epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by:
• Total Diagnosed Prevalent Population of Gene therapy in Ophthalmology
• Type-Specific cases of Gene therapy in Ophthalmology
• Gender-Specific cases of Gene therapy in Ophthalmology
• Age-Specific cases of Gene therapy in Ophthalmology
• Site-specific cases of Gene therapy in Ophthalmology
Download the report to understand which factors are driving gene therapy in ophthalmology epidemiology trends @ Gene Therapy in Ophthalmology Epidemiology Forecast
Gene Therapy in Ophthalmology Drugs Uptake and Pipeline Development Activities
The Gene Therapy in Ophthalmology drugs uptake section examines the rate at which newly launched or upcoming potential drugs are being adopted in the Gene Therapy in Ophthalmology market during the study period. This analysis covers drug uptake, patient adoption of therapies, and the sales performance of each drug.
Additionally, the therapeutics assessment section highlights the drugs with the most rapid uptake, shedding light on the factors driving their widespread use. It also provides a comparative analysis of these drugs based on their market share.
The report further delves into the Gene Therapy in Ophthalmology pipeline development activities, offering key insights into various therapeutic candidates in different stages of development and the major companies behind these innovations. It also covers recent collaborations, acquisitions, mergers, licensing agreements, patent details, and other critical information related to emerging therapies.
Gene Therapy in Ophthalmology Market Outlook
The gene therapy field faced setbacks between 1989 and 2012, but gained momentum after the EU approved the first gene therapy in 2012. This breakthrough spurred growth, with increasing investment and a rising number of early-stage candidates targeting previously untreatable chronic and genetic diseases.
In December 2017, Spark Therapeutics’ LUXTURNA became the first FDA-approved gene therapy for a genetic disease and the first for an inherited retinal disorder, marking a major milestone in ophthalmology. Following its approval, numerous companies entered the space, targeting genes linked to conditions like Retinitis Pigmentosa, LCA, LHON, Achromatopsia, Choroideremia, AMD, Diabetic Retinopathy, and DME.
Among emerging therapies, Beacon’s AGTC-501 is being developed for X-linked Retinitis Pigmentosa, with promising Phase II SKYLINE trial data shared in December 2023. For Diabetic Retinopathy and DME, the pipeline remains limited, with notable candidates including AbbVie/REGENXBIO’s ABBV-RGX-314 and 4D Molecular Therapeutics’ 4D-150.
Gene Therapy in Ophthalmology Market Strengths
• The eye is anatomically well-suited for gene therapy due to its small size, immune-privileged environment, and ease of local administration. This minimizes systemic exposure and reduces the risk of immune reactions, making ocular conditions a favorable starting point for gene therapy innovation.
• Following the success of Luxturna, the ophthalmic gene therapy pipeline has rapidly expanded with candidates targeting both rare inherited retinal disorders and more prevalent diseases like AMD. Regulatory agencies are increasingly supportive, providing orphan drug designations and fast-track reviews, which accelerate development timelines.
Gene Therapy in Ophthalmology Market Weaknesses
• The cost of gene therapy remains prohibitively high, limiting widespread adoption and reimbursement. Payers and healthcare systems face challenges in evaluating long-term value for single-dose, potentially curative treatments.
• Many therapies are still in early or mid-stage trials, with a lack of long-term real-world evidence on durability, safety, and retreatment potential. This creates uncertainty for clinicians, patients, and investors alike, especially for chronic or progressive conditions.
Scope of the Gene Therapy in Ophthalmology Market Report
• Study Period: 2020–2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Gene Therapy in Ophthalmology Companies: Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, GenSight Biologics, Neuropht Therapeutics, Adverum Biotechnologies, EyeVensys, 4D Molecular Therapeutics, Exegenesis Bio, MeiraGTx, Johnson & Johnson Innovative Medicine, Neuropht Therapeutics, 4D Molecular Therapeutics, Atsena Therapeutics, Ocugen, AbbVie, REGENXBIO, Skyline Therapeutics, Huida Gene Therapeutics, Opus Genetics, and others.
• Key Gene Therapy in Ophthalmology Therapies: BOTARETIGENE SPAROPARVOVEC, AGTC-501, MCO-010, GS030, 4D 125, CTX PDE6B, OCU400, BS01, LUMEVOQ, NR082 (NFS-01), ADVM-022 (AAV.7M8-AFLIBERCEPT), JNJ-1887 (AAVCAGSCD59/JNJ-81201887), EYS 606, 4D 150, EXG102-031, AAV-RPE65, AAV-CNGB3, AAV-CNGA3, NFS-02, 4D 110, ATSN-101, ATSN-201, OCU410, OCU410ST, AGTC-402, AGTC-401, ABBV-RGX-314, SKG0106, HG 004, OPGX-001, and others.
• Gene Therapy in Ophthalmology Therapeutic Assessment: Gene Therapy in Ophthalmology, currently marketed, and Gene Therapy in Ophthalmology emerging therapies
• Gene Therapy in Ophthalmology Market Dynamics: Gene Therapy in Ophthalmology market drivers and Gene Therapy in Ophthalmology market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
• Gene Therapy in Ophthalmology Unmet Needs, KOL’s views, Analyst’s views, Gene Therapy in Ophthalmology Market Access and Reimbursement
To learn more about the key players and advancements in the Gene Therapy in Ophthalmology treatment landscape, visit the Gene Therapy in Ophthalmology Market Analysis Report
Table of Contents
1. Gene Therapy in Ophthalmology Market Report Introduction
2. Executive Summary for Gene Therapy in Ophthalmology
3. SWOT analysis of Gene Therapy in Ophthalmology
4. Gene Therapy in Ophthalmology Patient Share (%) Overview at a Glance
5. Gene Therapy in Ophthalmology Market Overview at a Glance
6. Gene Therapy in Ophthalmology Disease Background and Overview
7. Gene Therapy in Ophthalmology Epidemiology and Patient Population
8. Country-Specific Patient Population of Gene Therapy in Ophthalmology
9. Gene Therapy in Ophthalmology Current Treatment and Medical Practices
10. Gene Therapy in Ophthalmology Unmet Needs
11. Gene Therapy in Ophthalmology Emerging Therapies
12. Gene Therapy in Ophthalmology Market Outlook
13. Country-Wise Gene Therapy in Ophthalmology Market Analysis (2020–2034)
14. Gene Therapy in Ophthalmology Market Access and Reimbursement of Therapies
15. Gene Therapy in Ophthalmology Market Drivers
16. Gene Therapy in Ophthalmology Market Barriers
17. Gene Therapy in Ophthalmology Appendix
18. Gene Therapy in Ophthalmology Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
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