Gene Therapies in Ophthalmology Market to Register Immense Growth at a CAGR of 57.8% by 2034 | DelveInsight

According to DelveInsight's analysis, the growth of the gene therapies market in ophthalmology is expected to be mainly driven by the increasing prevalence of eye disease, rise in awareness and access to treatment, increased adoption of enhanced genetic testing and robust pipeline activity for eye disease in the 7MM. 

LAS VEGAS, April 4, 2024 /PRNewswire/ -- DelveInsight's Gene Therapies in Ophthalmology Market Insights report includes a comprehensive understanding of current treatment practices, emerging gene therapy in ophthalmology, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].

Key Takeaways from the Gene Therapies in Ophthalmology Market Report

• According to DelveInsight's analysis, the market size for gene therapies in ophthalmology reached roughly USD 132 million in 2023 across the 7MM.
• The ophthalmology segment has emerged as the major therapeutic area targeted for the current clinical development of gene therapies, with indications including Retinitis Pigmentosa, Age-Related Macular Degeneration (AMD), Achromatopsia, Leber Congenital Amaurosis, Leber's Hereditary Optic Neuropathy (LHON), Stargardt Disease, and others. DelveInsight's analysis reveals that out of all the rare indications in ophthalmology, Retinitis Pigmentosa has the highest rate of gene therapies development. Furthermore, Wet-AMD, Geographic atrophy, and Diabetic Retinopathy have the largest prevalent pool among the target indications for gene therapies in ophthalmology. 
• Leading gene therapy in ophthalmology companies such as BEACON THERAPEUTICS, NANOSCOPE THERAPEUTICS, COAVE THERAPEUTICS, BIONIC SIGHT, GENSIGHT BIOLOGICS, ADVERUM BIOTECHNOLOGIES, EYEVENSYS, 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 are developing novel gene therapy in ophthalmology that can be available in the gene therapy in ophthalmology market in the coming years.
• The promising gene therapy in ophthalmology in the pipeline include Botaretigene sparoparvovec, AGTC-501, MCO-010, GS030, 4D-125, BS01, CTx-PDE6b, ATSN-101, LUMEVOQ (GS010), NR082 (NFS-01), ABBV-RGX-314, AAV-CNGB3, AGTC-401, JNJ-1887 (HMR-59), 4D-150, Ixo-vec (ADVM-022), and others.
• 4D Molecular Therapeutics is anticipating a Phase II dose expansion interim landmark data analysis of the PRISM study of 4D-150 in wet AMD at the Angiogenesis, Exudation, and Degeneration 2024 Conference and an update on the US Food and Drug Administration (FDA) feedback on Phase III pivotal trial plan in February 2024. 4D Molecular Therapeutics is also anticipating initial interim landmark data analysis for the Phase II population extension arm of 4D-150 in diabetic macular edema (DME) in the second half of 2024.
• In December 2023, the US FDA granted the regenerative medicine advanced therapy (RMAT) designation, and in October 2023, the European Medicines Agency (EMA) granted PRIority MEdicines (PRIME) designation to 4D-150 for intravitreal treatment of wet AMD.
• In December 2023, Ocugen announced that the US FDA had granted RMAT designation to OCU400 for the treatment of retinitis pigmentosa associated with RHO mutations.

Discover which therapies are expected to grab the major gene therapy in ophthalmology market share @ Gene Therapy in Ophthalmology Market Report

Gene Therapy in Ophthalmology Overview

Gene therapy is becoming a promising solution for retinal degenerative illnesses, particularly due to the retina's suitability for studying and treating eye conditions. Remarkably, it has pioneered approved gene therapy for inherited disorders in the United States, marking a significant advancement in medical treatment. Gene therapy presents a distinctive chance to address and potentially cure retinal degenerative illnesses, bringing optimism to millions impacted by genetic disorders or carrying mutations causing disease. Nevertheless, numerous hurdles must be overcome before gene therapy can be widely accessible for ocular conditions. Key areas such as determining the best timing for intervention, establishing standardized methods for evaluating outcomes, managing inflammation, raising awareness, and ensuring fair access are crucial for progressing treatments for inherited retinal diseases and transforming the outlook for visual impairment.

Gene Therapies in Ophthalmology Epidemiology Segmentation

In the US, the highest number of diagnosed prevalent cases comprised of diabetic retinopathy with approximately 5.1 million, while the lowest cases comprised of LHON in 2023.

In EU4 and the UK, Germany accounted for the highest number of retinitis pigmentosa-treated cases with approximately 24K in 2020.

The gene therapies in ophthalmology market report proffer epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Diagnosed Prevalent Cases of Selected Indications for Gene Therapies in Ophthalmology
• Eligible Cases of Selected Indications for Gene Therapies 
• Treated Cases of Selected Indications for Gene Therapies 

Gene Therapies in Ophthalmology Treatment Market 

In recent years, gene therapy has garnered significant attention in research, especially concerning its application in ocular treatments. The eye is notable among the body's organs as it has gained approval from the FDA for gene therapy, distinguishing it from others in this regard. LUXTURNA, also referred to as voretigene neparvovec-rzyl, is a singular genetic therapy designed to address vision loss caused by a genetic anomaly in both instances of the RPE65 gene. The FDA approved Spark Therapeutics' LUXTURNA in December 2017, while the European Commission (EU) followed suit in November 2018. This authorization extends to all 28 EU member states, as well as Iceland, Liechtenstein, and Norway. The approval of LUXTURNA for individuals diagnosed with inherited retinal dystrophy due to biallelic RPE65 mutations, where there are still functional retinal cells, marks a significant advancement in the progress of ocular gene therapy.

The main treatment for AMD and DME entails administering injections of anti-VEGF. Vision decline in both ailments arises from unregulated expansion and fluid seepage of retinal blood vessels. Anti-VEGF treatments work by obstructing the overgrowth of these vessels. Current clinical studies are investigating the potential of introducing genes into retinal cells. This method seeks to prompt cells to independently manufacture anti-VEGF, effectively turning retinal cells into small-scale production units for needed proteins. The overarching goal is to reduce the need for frequent injections during treatment.

Researchers remain deeply engaged in the field, yet concerns persist regarding potential immune and inflammatory reactions that could undermine the effectiveness or safety of treatments. The eyes, being particularly prone to inflammation, are especially vulnerable to such issues. The severity of these reactions depends largely on the choice of vector and how it is administered. Various preclinical and clinical investigations have highlighted a link between the dosage of the vector and the level of immune response and inflammation in the eye. Moreover, the method of administration directly impacts the intensity of these reactions. For example, subretinal delivery tends to elicit a less pronounced immune response compared to intravitreal injection. Nevertheless, studies have suggested that subretinal delivery may trigger a more vigorous inflammatory response.

To know more about gene therapy in ophthalmology, visit @ Gene Therapies in Ophthalmology Analysis

Gene Therapies in Ophthalmology Pipeline Therapies and Key Companies

• BOTARETIGENE SPAROPARVOVEC: JOHNSON & JOHNSON INNOVATIVE MEDICINE /MEIRAGTX
• AGTC-501: BEACON THERAPEUTICS
• MCO-010: NANOSCOPE THERAPEUTICS
• GS030: GENSIGHT BIOLOGICS
• 4D 125: 4D MOLECULAR THERAPEUTICS
• CTX PDE6B: COAVE THERAPEUTICS
• OCU 400: OCUGEN
• BS01: BIONIC SIGHT
• LUMEVOQ: GENSIGHT BIOLOGICS
• NR082 (NFS-01): NEUROPHTH THERAPEUTICS
• ADVM-022 (AAV.7M8-AFLIBERCEPT): ADVERUM BIOTECHNOLOGIES
• JNJ-1887 (AAVCAGSCD59/JNJ-81201887): JOHNSON & JOHNSON INNOVATIVE MEDICINE
• EYS 606: EYEVENSYS
• 4D 150: 4D MOLECULAR THERAPEUTICS
• EXG102-031: EXEGENESIS BIO
• AAV-RPE65: MEIRAGTX/JOHNSON & JOHNSON INNOVATIVE MEDICINE
• AAV-CNGB3: MEIRAGTX/JOHNSON & JOHNSON INNOVATIVE MEDICINE
• AAV-CNGA3: MEIRAGTX/JOHNSON & JOHNSON INNOVATIVE MEDICINE
• NFS-02: NEUROPHTH THERAPEUTICS
• 4D 110: 4D MOLECULAR THERAPEUTICS
• ATSN-101: ATSENA THERAPEUTICS
• ATSN-201: ATSENA THERAPEUTICS
• OCU410: OCUGEN
• OCU410ST: OCUGEN
• AGTC-402: BEACON THERAPEUTICS
• AGTC-401: BEACON THERAPEUTICS
• ABBV-RGX-314: ABBVIE/REGENXBIO
• SKG0106: SKYLINE THERAPEUTICS
• HG 004: HUIDAGENE THERAPEUTICS
• OPGX-001: OPUS GENETICS

Learn more about the FDA-approved gene therapy in ophthalmology @ Gene Therapies in Ophthalmology Treatment 

Gene Therapies in Ophthalmology Market Dynamics

The dynamics of gene therapy in ophthalmology market are expected to change in the coming years. AAVs have emerged as the predominant vectors for delivering genes of interest to target tissues with improved specificity, efficiency, and safety, leading companies to offer innovative payment plans for one-time gene therapies, which can enhance the uptake of gene therapies. Additionally, larger firms, leveraging their resources and expertise, have gained an edge in the gene therapy industry by organizing intricate development pathways. The exponential advancements in the field of gene therapy for eye disease over the past few decades hold the potential to treat most eye diseases caused by genetic mutations.

Furthermore, many potential gene therapies are being investigated, and it is safe to predict that the treatment space will significantly impact gene therapy in ophthalmology market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of gene therapy in ophthalmology market in the 7MM.

However, several factors may impede the growth of gene therapy in ophthalmology market. Even when a rare disease therapy is available, the out-of-pocket cost to patients can be prohibitive, as orphan drugs are priced higher than common treatments to generate sufficient returns on investment despite small patient populations. Simply delivering the product to the desired tissue can be a physical challenge due to the isolated nature of the eyes and the delicate nature of diseased tissue, while gene therapies may face more rivalry for production capacity as more companies join the market, potentially increasing prices or reducing its utility as a treatment choice. A potential crisis with gene therapies for rare disorders is looming in Europe, as many companies have failed to secure reimbursement in the EU and withdrawn the drug after approval.

Moreover, gene therapy in ophthalmology treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the gene therapy in the ophthalmology market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact gene therapy in ophthalmology market growth.

Of the target indications, Retinitis Pigmentosa and Wet AMD are anticipated to generate the most revenue by 2034 due to the highest level of clinical development and authorization of gene therapies. Apart from AMD, ocular diseases associated with diabetes may offer the most commercial potential for gene therapy because millions of people with diabetes suffer from ocular issues. Among them include diabetic retinopathy and diabetic macular edema.

Gene Therapies in Ophthalmology Market Report Metrics	Details
Study Period	2020–2034
Coverage	7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].
Gene Therapy in Ophthalmology Market CAGR (2020–2034)	57.8 %
Gene Therapy in Ophthalmology Market Size in 2020	USD 50 Million
Key Gene Therapy in Ophthalmology Companies	BEACON THERAPEUTICS, NANOSCOPE THERAPEUTICS, COAVE THERAPEUTICS, BIONIC SIGHT, GENSIGHT BIOLOGICS, ADVERUM BIOTECHNOLOGIES, EYEVENSYS, 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 Pipeline Gene Therapies in Ophthalmology	BOTARETIGENE SPAROPARVOVEC, AGTC-501, MCO-010, GS030, 4D 125, CTX PDE6B, OCU 400, 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, NFS-02, 4D 110, ATSN-101, OCU410, OCU410ST, AGTC-401, ABBV-RGX-314, SKG0106, HG 004, OPGX-001, and others

 

Scope of the Gene Therapies in Ophthalmology Market Report

• Therapeutic Assessment: Gene Therapy in Ophthalmology current marketed and emerging therapies
• Gene Therapies in Ophthalmology Market Dynamics: Key Market Forecast Assumptions of Emerging Gene Therapy in Ophthalmology Drugs, Conjoint Analysis, and Market Outlook
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL's views, Analyst's views, Gene Therapy in Ophthalmology Market Access and Reimbursement

Discover more about gene therapy in ophthalmology in development @ Gene Therapy in Ophthalmology Clinical Trials

Table of Contents

1.	Gene Therapies in Ophthalmology Key Insights
2.	Gene Therapies in Ophthalmology Report Introduction
3.	Gene Therapies in Ophthalmology Executive Summary
4.	Gene Therapies in Ophthalmology Key Events
5	Gene Therapies in Ophthalmology Epidemiology and Market Forecast Methodology
6	Gene Therapies In Ophthalmology Market Overview at a Glance in the 7MM
6.	Gene Therapies in Ophthalmology Disease Background And Overview
7.	Gene Therapies in Ophthalmology Epidemiology And Patient Population
8.	Gene Therapies in Ophthalmology Marketed Drugs
9.	Gene Therapies in Ophthalmology Emerging Drugs
10.	Gene Therapies in Ophthalmology: 7MM Analysis
11.	Unmet Needs
12.	SWOT Analysis
13.	KOL Views
14.	Market Access and Reimbursement
15.	Appendix
16.	DelveInsight Capabilities
17.	Disclaimer
18.	About DelveInsight

 

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