Gene therapy in ophthalmology Market: Epidemiology, Therapies, Companies, DelveInsight | Johnson & Johnson Innovative Medicine, Meiragtx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4d Molecular Therapeutics, Coave Therapeutics, Bioni

Gene therapy in ophthalmology emerging therapies, such as RGX-314, GS010, NFS-01, and others, are expected to boost the Gene therapy in ophthalmology Market in the upcoming years.

DelveInsight has launched a new report on "Gene therapy in ophthalmology - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Gene therapy in ophthalmology, historical and forecasted epidemiology as well as the Gene therapy in ophthalmology market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

Discover about the Gene therapy in ophthalmology market report @ https://www.delveinsight.com/report-store/gene-therapy-in-ophthalmology-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Some of the key facts of the Gene therapy in ophthalmology Market Report:

The Gene Therapy in Ophthalmology market size across the 7MM was approximately USD 50 million in 2020 and is expected to expand throughout the forecast period (2024-2034).
According to DelveInsight, in 2020, the Gene Therapy in Ophthalmology market in the US was the largest among the 7MM, with a market size of approximately USD 35 million, and it is projected to grow by 2034.
Currently, LUXTURNA is the only approved gene therapy for ophthalmology, having received FDA approval in December 2017 and EU approval in November 2018.
In 2020, the highest number of prevalent cases across the selected ophthalmic conditions in the 7MM was for Diabetic Retinopathy, followed by Wet-AMD, with the fewest cases being for Leber Hereditary Optic Neuropathy.
For treated cases across the 7MM in 2020, Achromatopsia had the lowest number of cases.
In December 2023, the US FDA granted RMAT designation to 4D-150 for the intravitreal treatment of Wet AMD, and in October 2023, the EMA granted PRIME designation to the same therapy. 4D Molecular Therapeutics is expecting interim data from the Phase II dose expansion of the PRISM study of 4D-150 at the Angiogenesis, Exudation, and Degeneration 2024 Conference, and a Phase III trial plan update in February 2024.
In 2020, Diabetic Retinopathy accounted for the highest number of diagnosed prevalent cases among selected gene therapy indications in ophthalmology, with approximately 9.2 million cases across the 7MM. In the US, Diabetic Retinopathy also had the highest number of diagnosed cases, totaling around 4.8 million, while Leber Hereditary Optic Neuropathy had the fewest cases during the same year.
Additionally, in December 2023, Ocugen announced that the US FDA granted RMAT designation to OCU400 for treating retinitis pigmentosa associated with RHO mutations.
In March 2025, The FDA approved Encelto (revakinagene taroretcel-lwey) for macular telangiectasia type 2 (MacTel). This implantable cell therapy delivers ciliary neurotrophic factor to the retina, aiming to slow disease progression.
In December 2024, ViGeneron received FDA clearance for its investigational new drug (IND) application for VG801, an mRNA trans-splicing gene therapy designed to treat Stargardt disease and other ABCA4-linked retinal dystrophies.
Gene therapy companies in the ophthalmology field, such as Johnson & Johnson Innovative Medicine, Meiragtx, Beacon Therapeutics, Nanoscope Therapeutics, Gensight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, Neurophth Therapeutics, Adverum Biotechnologies, and Eyevensys, are advancing vision care by developing innovative treatments for inherited retinal diseases and other eye conditions.
Promising Gene therapy in ophthalmology therapies include RGX-314, GS010, NFS-01, and others.

Gene therapy in ophthalmology Overview

Gene therapy seeks to target the root cause of diseases, particularly genetic changes. It involves using genetic material, such as DNA or RNA, to treat or prevent illness. The delivered genetic material carries instructions to alter the production of specific proteins within the cell. In certain diseases, this may involve correcting the production of too much, too little, or incorrectly produced essential proteins. In ophthalmology, gene therapy is being explored for various conditions, including dry and wet age-related macular degeneration, Stargardt disease, retinitis pigmentosa, Leber's Congenital Amaurosis (LCA), and others. Ongoing clinical trials in ophthalmic gene therapy are transforming treatment methods and offering promising possibilities for treating inherited retinal diseases and restoring vision.

Gene therapy in ophthalmology Market Outlook

The gene therapy field saw a mix of progress and setbacks from 1989 to 2012. After two decades of unmet expectations, the approval of the first gene therapy in the EU in 2012 marked a turning point, leading to a significant increase in gene therapy development. These therapies are now seen as highly promising for treating chronic, disabling, and previously untreatable diseases. Manufacturers are investing more in this area, and the number of clinical development projects, especially in the early stages, is rising.

In December 2017, Spark Therapeutics announced that its drug candidate LUXTURNA received US FDA approval to treat patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. LUXTURNA became the first FDA-approved gene therapy for a genetic disease, the first and only pharmacologic treatment for an inherited retinal disease, and the first AAV vector gene therapy approved in the US. Following the approval of LUXTURNA, several companies entered the ophthalmology gene therapy market, targeting a variety of genetic diseases, including Retinitis Pigmentosa, Leber's Congenital Amaurosis (LCA), LHON, Achromatopsia, Choroideremia, AMD (wet and dry forms), Diabetic Retinopathy, DME, and more.

Among the emerging candidates for Retinitis Pigmentosa, Beacon's AGTC-501 seeks to restore RPGR gene function in patients with X-linked retinitis pigmentosa. In December 2023, Beacon presented three-month data from its randomized, controlled, multicenter Phase II SKYLINE trial of AGTC-501 at the FLORetina ICOOR 2023 conference. The therapeutic development landscape for Diabetic Retinopathy and DME remains limited, with only a few notable players like AbbVie/REGENXBIO's ABBV-RGX-314 and 4D Molecular Therapeutics' 4D-150.

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Gene therapy in ophthalmology Emerging Drugs
RGX-314: REGENXBIO
GS010: GenSight Biologics
NFS-01: Neurophth Therapeutics

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, Neurophth Therapeutics, Adverum Biotechnologies, Eyevensys, 4d Molecular Therapeutics, Exegenesis Bio, Meiragtx, Johnson & Johnson Innovative Medicine, Neurophth Therapeutics, 4d Molecular Therapeutics, Atsena Therapeutics, Ocugen, Abbvie, Regenxbio, Skyline Therapeutics, Huidagene Therapeutics, Opus Genetics, and others
Key Gene therapy in ophthalmology Therapies: RGX-314, GS010, NFS-01, and others
Gene therapy in ophthalmology Therapeutic Assessment: Gene therapy in ophthalmology current 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

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Key benefits of the Gene therapy in ophthalmology Market Report:
Gene therapy in ophthalmology market report covers a descriptive overview and comprehensive insight of the Gene therapy in ophthalmology Epidemiology and Gene therapy in ophthalmology market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
The Gene therapy in ophthalmology market report provides insights into the current and emerging therapies.
The Gene therapy in ophthalmology market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Gene therapy in ophthalmology market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Gene therapy in ophthalmology market.

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Table of Contents

1. Report Introduction
2. Executive Summary
3. SWOT analysis
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. 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. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Gene therapy in ophthalmology Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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Related Reports:

Gene therapy in ophthalmology Pipeline Insights, DelveInsight

"Gene therapy in ophthalmology Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Gene therapy in ophthalmology market. A detailed picture of the Gene therapy in ophthalmology pipeline landscape is provided, which includes the disease overview and Gene therapy in ophthalmology treatment guidelines.

Contact Info:

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info@delveinsight.com
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About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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