Gene Therapy in Ophthalmology Market Size in the 7MM was ~USD 50 million in 2020 and is projected to grow by 2034, estimates DelveInsight

DelveInsight's "Gene Therapy in Ophthalmology Market Insights, Epidemiology, and Market Forecast - 2034" report delivers an in-depth understanding of Gene Therapy in Ophthalmology, historical and forecasted epidemiology, as well as the Gene Therapy in Ophthalmology market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

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Key Takeaways from the Gene Therapy in Ophthalmology Market Report
• Among all the diagnosed prevalent cases of selected indications for gene therapies in ophthalmology, the highest cases comprised of Diabetic Retinopathy, with approximately 9,200,000 cases in the 7MM in 2020.
• In the US, the highest number of diagnosed prevalent cases comprised of Diabetic Retinopathy with approximately 4,800,000, while the lowest cases comprised Leber Hereditary Optic Neuropathy in 2020.
• In EU4 and the UK, Germany accounted for the highest number of Retinitis Pigmentosa-treated cases with approximately 24,000 in 2020.
• In Japan, the total number of wet-AMD-eligible cases was approximately 800,000 in 2020.
• The leading Gene Therapy in Ophthalmology Companies such as Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, Neurophth Therapeutics, Adverum Biotechnologies, Eyevensys, Exegenesis Bio, Atsena Therapeutics, Ocugen, AbbVie, Regenxbio, Skyline Therapeutics, Huidagene Therapeutics, Opus Genetics and others.
• Promising Gene Therapy in Ophthalmology Therapies such as RGX-314, GS010, NFS-01, and others.

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Gene Therapy in Ophthalmology Epidemiology Segmentation in the 7MM
• 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

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Gene Therapy in Ophthalmology Marketed Drugs
• LUXTURNA is an AAV vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells, as determined by the treating physician. Mutations in the RPE65 (retinal pigment epithelial 65 kDa protein) gene lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle and resulting in impairment of vision. Injection of LUXTURNA into the subretinal space results in the transduction of some retinal pigment epithelial cells with a cDNA encoding normal human RPE65 protein, thus providing the potential to restore the visual cycle. It was approved by the US FDA in December 2017, and by the European Commission in November 2018.

Gene Therapy in Ophthalmology Emerging Drugs
• RGX-314: REGENXBIO
RGX-314 is a one-time subretinal treatment that includes the NAV AAV8 vector containing a gene encoding for a monoclonal antibody fragment. The expressed protein is designed to neutralize vascular endothelial growth factor (VEGF) activity, modifying the pathway for the formation of new leaky blood vessels and retinal fluid accumulation. RGX-314 is being developed as a potentially one-time treatment for wet AMD, diabetic retinopathy, and other additional chronic retinal conditions treated with anti-VEGF. ATMOSPHERE, the first of two planned pivotal trials for the evaluation of subretinal delivery of RGX-314 in patients with wet AMD, is active and enrolling patients. In May 2023 the company announced the transfer of the IND applications to AbbVie for all ongoing gene therapy in ophthalmology clinical trials and the expansion of the ATMOSPHERE and ASCENT pivotal trials of RGX-314 delivered subretinally for the treatment of patients with wet AMD to support new global registration plans. The new global site plans and expanded enrollment targets are expected to support regulatory submissions of ABBV-RGX-314 with the US FDA and the EMA in late 2025 through the first half of 2026.

• GS010: GenSight Biologics
GS010 targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, which allows the platform to specifically address defects inside the mitochondria using an AAV vector. The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. Currently, the drug is in the Phase III stage of its development for the treatment of LHON.

• NFS-01: Neurophth Therapeutics
NFS-01 (NR082, rAAV2-ND4), a novel recombinant adeno-associated viral vector, serotype 2, containing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 (ND4) gene under the control of the cytomegalovirus promoter and enhancer, is a novel gene therapy product that is being developed for the treatment of LHON associated with mtND4 mutations. NR082 can deliver the correct genes to the patient's damaged optic ganglion cells through intravitreal injection, thus repairing the mitochondrial biological respiratory chain, and restoring the vitality and visual function of optic ganglion cells. Currently, the drug is in the Phase II/III stage of its development for the treatment of Leber's hereditary optic atrophy.

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Gene Therapy in Ophthalmology Companies
Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, Neurophth Therapeutics, Adverum Biotechnologies, Eyevensys, Exegenesis Bio, Atsena Therapeutics, Ocugen, AbbVie, Regenxbio, Skyline Therapeutics, Huidagene Therapeutics, Opus Genetics and others.

Gene Therapy in Ophthalmology Market Outlook
The gene therapy field experienced ups and downs between 1989 and 2012. After two decades of dashed expectations, the first gene therapy was approved in the EU in 2012. This likely boosted this field, and the development of gene therapies increased prominently. Gene therapies are considered very promising to treat many chronic and disabling previously untreatable diseases. Manufacturers are investing more in this field, and an increasing number of products are under clinical development, mostly in the early stages.

Gene Therapy in Ophthalmology Treatment Market
Gene therapy is gaining strong momentum within the ophthalmology treatment market, driven by its ability to target the genetic root causes of vision-threatening disorders rather than providing symptomatic relief. The eye is particularly well suited for gene-based treatments due to localized delivery, immune privilege, and clearly defined genetic mutations, especially in inherited retinal diseases such as retinitis pigmentosa, Leber congenital amaurosis, and choroideremia, along with select degenerative conditions like age-related macular degeneration.

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Scope of the Gene Therapy in Ophthalmology Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Gene Therapy in Ophthalmology Companies- Johnson & Johnson Innovative Medicine, MeiraGTx, Beacon Therapeutics, Nanoscope Therapeutics, GenSight Biologics, 4D Molecular Therapeutics, Coave Therapeutics, Bionic Sight, Neurophth Therapeutics, Adverum Biotechnologies, Eyevensys, Exegenesis Bio, Atsena Therapeutics, Ocugen, AbbVie, Regenxbio, Skyline Therapeutics, Huidagene Therapeutics, Opus Genetics and others.
• Gene Therapy in Ophthalmology Therapies- RGX-314, GS010, NFS-01, and others.
• Gene Therapy in Ophthalmology Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Gene Therapy in Ophthalmology Unmet Needs, KOL's views, Analyst's views, Gene Therapy in Ophthalmology Market Access and Reimbursement

Table of Contents
1: Key Insights
2: Gene Therapy in Ophthalmology Market Report Introduction
3: Gene Therapy in Ophthalmology Market Overview at a Glance
4: Gene Therapy in Ophthalmology Epidemiology and Market Methodology
5: Gene Therapy in Ophthalmology Executive Summary
6: Key Events
7: Gene Therapy in Ophthalmology Background and Overview
8: Gene Therapy in Ophthalmology Epidemiology and Patient Population
9: Gene Therapy in Ophthalmology Patient Journey
10: Gene Therapy in Ophthalmology Marketed drugs
11: Gene Therapy in Ophthalmology Emerging Drugs
12. Key Cross Competition
13: Gene Therapy in Ophthalmology Unmet Needs
14: Gene Therapy in Ophthalmology SWOT Analysis
15: Key Opinion Leaders' Views
16: Reimbursement and Market Access
17: Appendix
18: DelveInsight Capabilities
19: Disclaimer
20: About DelveInsight

About Us

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This release was published on openPR.