Retinal Gene Therapy Market Snapshot

According to Future Market Insights research, during the projected period, the global retinal gene therapy market is expected to grow at a CAGR of 9.2%. The market value is projected to increase from USD 97.3 Million in 2023 to USD 235.4 Million by 2033. The retinal gene therapy market was valued at USD 89.9 Million at the end of 2022 and is anticipated to exhibit Y-o-Y growth of 8.2% in 2023.

Market Outlook:

Data Points Market Insights
Market Value 2022 USD 89.9 Million
Market Value 2023 USD 97.3 Million
Market Value 2023 USD 235.4 Million
CAGR 2023 to 2033 9.2%
Share of Top 5 Countries 55.7%
Key Players Gyroscope Therapeutics (Novartis); Applied Genetic Technologies Corporation (AGTC); Spark Therapeutics; Adverum Biotechnologies; REGENXBIO Inc.; Biogen Inc.; Nightstar Therapeutics; MeiraGTx Holdings plc; GenSight Biologics; RetroSense Therapeutics; Second Sight Medical Products; Oxford Biomedica; Hemera Biosciences; Adverio Pharmaceuticals

Retinal gene therapy is a treatment approach that aims to restore or replace the faulty or missing genes that cause inherited retinal diseases. Inherited retinal diseases like Retinitis Pigmentosa or Leber Congenital Amaurosis occur due to a genetic mutation that leads to the degeneration of photoreceptor cells in the retina, ultimately leading to vision loss or blindness.

Retinal gene therapy is a promising approach as it has the potential to provide a one-time treatment that could help patients with inherited retinal diseases to maintain or restore their vision. However, this treatment is still in the early stages of development and is not yet available as a standard treatment.

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Sales Analysis of Retinal Gene Therapy Market from 2017 to 2022 Vs Market Outlook for 2023 to 2033

Sales of the retinal gene therapy grew at a CAGR of 7.7% between 2017 and 2022. The global market for retinal gene therapy contributes around 1.3% to the USD 7.0 Billion gene therapy market in 2022.

The prevalence of inherited retinal diseases like Retinitis pigmentosa, Leber Congenital Amaurosis, and Choroideremia is increasing globally. This rise in the prevalence of these diseases is driving demand for new treatment options.

  • As per an article published in NCBI, 2023, around one in 5000 people worldwide are carriers of non-syndromic retinitis pigmentosa, which has a prevalence rate of one in 1000. Men are impacted slightly more often than women because men express the X-linked variant more frequently.
  • According to an article reported in the American Academy of Ophthalmology, 2022, One in 50,000 to one in 100,000 people are believed to be affected by the unusual chorioretinal dystrophy known as choroideremia. The dystrophy typically affects men because of its X-linked etiology, while it can also affect women in rare cases or as asymptomatic carriers.

There is currently no cure for inherited retinal diseases. Existing treatments focus mainly on slowing disease progression, but they do not halt or reverse the damage already done. Retinal gene therapy has the potential to offer a once-in-a-lifetime cure or long-term solution for these debilitating diseases. As such, this significant unmet need is a key driver of continued development and commercialization efforts.

Significant advancements in surgical methods required for delivering altered genes to the retinal tissue have been made over the last two decades, which has facilitated the development of novel and revolutionary treatment approaches.

  • Unlike traditional treatments like medications or surgery, retinal gene therapy aims at correcting the underlying genetic defect in the patient. This approach has the potential to provide a one-time cure that can halt, or in some cases possibly restore, vision loss permanently.
  • Certain gene therapies in development are designed as low frequency treatments designed to limit the number of administrations beyond the initial treatment. The success of Luxturna has shown the potential of low frequency therapies in the field.

Owing to such factors, FMI projects the global market to grow over a CAGR of 9.2% through the forecast years.

What are the Key Opportunities in the Market?

The technological advancements in gene editing, viral vector development and delivery systems are enabling the development of more efficient and safe gene-based therapies. This has led to a surge in research and development activities aimed at exploring the potential of gene therapies for inherited retinal diseases.

  • Gene editing technologies like CRISPR/Cas9 and Zinc Finger Nucleases (ZFNs) have enabled the precise and efficient editing of DNA, allowing for specific genes to be targeted and corrected.

Viral vectors, which are used to deliver genes into target cells, are key to gene therapy. The development of safer and more efficient viral vectors has been a major area of focus for gene therapy. For example, Adeno-associated virus (AAV) vectors have become the most commonly used viral delivery system for genetic therapies in humans.

  • For example, Adeno-associated virus (AAV) vectors have become the most commonly used viral delivery system for genetic therapies in humans. AAV vectors have been underpinning several FDA-approved gene therapies including Luxturna, a one-time gene therapy for the treatment of genetic vision loss, showing the potential of this vector technology.

Governments and private entities worldwide are increasingly investing in gene therapies for inherited diseases, including retinal disease. This funding increase has encouraged continued research and development, accelerating the development of gene therapies while providing financial support for clinical trials and commercialization efforts.

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Which are the Potential Factors Capable of Restraining the Growth of Global Market?

The probable obstruction to growth is affordability, as the cost associated with multiple dosages is one of the major concerns for patients and caregivers, as it would involve high costs and reimbursement procedures.

There remains regulatory uncertainty around reimbursement and market access for gene therapy products which also hinders growth, with potential regulatory challenges associated with gene therapies, affordability, access by health systems, and access to specialized care providers affecting the commercial uptake.

Retinal gene therapy being an early-phase developmental technology, the development process involves significant research and development costs, which require immense investments of time, money and resources.

The number of potential patients with inherited retinal diseases is limited, meaning the market size for these treatments is small. Also, awareness among healthcare professionals regarding this therapy is still limited in several low- to middle-income economic countries.

Inherently there are concerns surrounding gene therapy safety even after approval whereby follow up must take place due to potential side effects that may emerge.

Overall, these factors are capable of restraining the expansion of the market in the future as well.

Country-wise Insights

What Makes the USA a Dominating Market in the Retinal gene therapy Industry?

The USA contributed the most in the North America and made a profitable market, holding about 34.2% in the global market by the end of year 2022.

Retinal gene therapy research in the USA is being conducted by many renowned research institutions and companies, which have made significant progress in the field over the last decade. These technological advancements have given the USA market a leading edge in Retinal gene therapy development.

Likewise, the USA government also provides significant funding towards research and development in the area of gene therapy, with a major portion of this going towards Retinal gene therapy.

What is Making Germany to Grow Significantly in the European Market?

Germany is one of the leading countries in Europe, with a significant growth rate of 7.5% over the forecast period.

Germany, together with the United Kingdom and France, has the highest incidence of age-related macular degeneration (AMD)-related blindness and also has a large patient pool inflicted with inherited retinal diseases. This patient pool is likely to create demands for gene therapies for the treatment of their visual abnormalities, consequently, this may provide companies to have a sizeable and established market for their products.

How China is Emerging as a Prominent Market in the Global Industry?

China is projected to be the most attractive market in the East Asia market, accounting for the global market share of around 6.5% in 2022.

With significant advances in gene therapy manufacturing technology, China is taking advantage of its affordable domestic manufacturing of gene therapy.

Many Chinese domestic companies are research and development focused on the development of gene therapies for inherited retinal diseases in the country. The Chinese government offers significant support to life science research, development, and production, which has led to a surge in domestic research and development within the country.

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Category-wise Insights

Which Segment Has Dominated the Indication category?

The Stargardt disease accounted for the dominant share of 32.8% in the global market in 2022 and expected to expand at a CAGR of 9.7% throughout the forecast period.

The growing prevalence of Stargardt disease globally, particularly in developed economies, has created high demand for retinal gene therapy worldwide. The potential to restore visual function in patients with inherited retinal diseases like Stargardt disease has led to a surge in research and development in the area, with many companies working on retinal gene therapy to help address the high unmet demand for innovative treatments.

Which End User Held the Major Chunk of the Market?

Hospitals held the significant share in the global market as a distribution channel with holding about 43.6% of the overall market by the end of 2022.

Retinal gene therapy is a highly technical procedure and requires specialization and expertise in delivering the therapy, making hospitals with specialized care facilities a natural fit as the leading end-users of the therapy.

Hospitals have the advantage of direct patient access which can be crucial for high-end gene therapies where patients require hospitalization to deliver the treatment. Retinal gene therapy could require such specialized inpatient care before the patient is transitioned to at-home care.

Competitive Landscape

Due to the high market concentration and sub-segmented market, companies are relying on research and development of novel therapies to deliver the treatment more effectively and efficiently. Companies such as Janssen Biotech, have currently entered the market and continue to invest heavily in research and development.

Overall, research and development, strategic collaborations, regulatory approvals, and international expansion remain top priorities for companies looking to increase their market share in the retinal gene therapy industry, with competition driving innovation, and making the therapy available and more cost-effective on a wider scale.

For instance

  • In December 2021, Ocugen, Inc. announced that the USA FDA approved its application for an investigational new drug to start a phase 1/2 clinical evaluation for the gene therapy potential candidate OCU400 for the treatment of inherited retinal degeneration.
  • In October 2021, GenSight Biologics announced that the FDA granted GS030 optogenetic therapy for the medical treatment of retinitis pigmentosa fast track status.

Similarly, recent developments have been tracked by the team at Future Market Insights related to companies in the retinal gene therapy market, which are available in the full report.

Report Scope as per Retinal Gene Therapy Industry Analysis

Attribute Details
Forecast Period 2023 to 2033
Historical Data Available for 2017 to 2022
Market Analysis USD Million for Value
Key Regions Covered North America; Latin America; Europe; South Asia; East Asia; Oceania; Middle East & Africa
Key Countries Covered USA, Canada, Brazil, Mexico, Argentina, Germany, UK, France, Italy, Spain, Russia, BENELUX, China, Japan, South Korea, India, Thailand, Indonesia, Malaysia, Australia, New Zealand, Türkiye, South Africa, GCC Countries, Northern Africa
Key Segments Covered Indication, End User, and Region
Key Companies Profiled Gyroscope Therapeutics (Novartis); Applied Genetic Technologies Corporation (AGTC); Spark Therapeutics; Adverum Biotechnologies; REGENXBIO Inc.; Biogen Inc.; Nightstar Therapeutics; MeiraGTx Holdings plc; GenSight Biologics; RetroSense Therapeutics; Second Sight Medical Products; Oxford Biomedica; Hemera Biosciences; Adverio Pharmaceuticals
Report Coverage Market Forecast, Competition Intelligence, DROT Analysis, Market Dynamics and Challenges, Strategic Growth Initiatives
Customization & Pricing Available upon Request
Table of Content
  • 1. Executive Summary
  • 2. Market Overview
  • 3. Market Trends
  • 4. Value Added Insights
  • 5. Market Background
  • 6. Global Market Demand (in Value or Size in USD Million) Analysis 2017 to 2022 and Forecast, 2023 to 2033
  • 7. Global Market Analysis 2017 to 2022 and Forecast 2023 to 2033, By Indication
    • 7.1. Leber Congenital Amaurosis (LCA)
    • 7.2. Stargardt Disease
    • 7.3. Choroideremia
    • 7.4. X-linked Retinitis Pigmentosa (RP)
    • 7.5. Others
  • 8. Global Market Analysis 2017 to 2022 and Forecast 2023 to 2033, By End User
    • 8.1. Hospitals
    • 8.2. Academic & Research Institutes
    • 8.3. Ophthalmic Clinics
    • 8.4. Others
  • 9. Global Market Analysis 2017 to 2022 and Forecast 2023 to 2033, by Region
    • 9.1. North America
    • 9.2. Latin America
    • 9.3. Europe
    • 9.4. South Asia
    • 9.5. East Asia
    • 9.6. Oceania
    • 9.7. Middle East & Africa
  • 10. North America Market Analysis 2017 to 2022 and Forecast 2023 to 2033
  • 11. Latin America Market Analysis 2017 to 2022 and Forecast 2023 to 2033
  • 12. Europe Market Analysis 2017 to 2022 and Forecast 2023 to 2033
  • 13. East Asia Market Analysis 2017 to 2022 and Forecast 2023 to 2033
  • 14. South Asia Market Analysis 2017 to 2022 and Forecast 2023 to 2033
  • 15. Oceania Market Analysis 2017 to 2022 and Forecast 2023 to 2033
  • 16. Middle East & Africa Market Analysis 2017 to 2022 and Forecast 2023 to 2033
  • 17. Market Structure Analysis
  • 18. Competition Analysis
    • 18.1. Gyroscope Therapeutics (Novartis)
    • 18.2. Applied Genetic Technologies Corporation (AGTC)
    • 18.3. Spark Therapeutics
    • 18.4. Adverum Biotechnologies
    • 18.5. REGENXBIO Inc.
    • 18.6. Biogen Inc.
    • 18.7. Nightstar Therapeutics
    • 18.8. MeiraGTx Holdings plc
    • 18.9. GenSight Biologics
    • 18.10. RetroSense Therapeutics
    • 18.11. Second Sight Medical Products
    • 18.12. Oxford Biomedica
    • 18.13. Hemera Biosciences
    • 18.14. Adverio Pharmaceuticals
  • 19. Assumptions and Acronyms Used
  • 20. Research Methodology

Key Market Segments Covered in Retinal Gene Therapy Market Industry Analysis

By Indication:

  • Leber Congenital Amaurosis (LCA)
  • Stargardt Disease
  • Choroideremia
  • X-linked Retinitis Pigmentosa (RP)
  • Others

By End User:

  • Hospitals
  • Academic & Research Institutes
  • Ophthalmic Clinics
  • Others

By Region:

  • North America
  • Latin America
  • Europe
  • South Asia
  • East Asia
  • Oceania
  • Middle East and Africa

Frequently Asked Questions

How Big is the Retinal Gene Therapy Market?

The market is valued at USD 97.3 million in 2023.

How Big will the Retinal Gene Therapy Market be by 2033?

The market is estimated to reach USD 235.4 million by 2033.

Which Countries Dominate the Global Market?

The United States, Japan, and China dominate the global market.

What is the Growth Forecast for Retinal Gene Therapy Market?

The market is forecast to register a CAGR of 9.2% through 2033.

What are the Key Opportunities in the Retinal Gene Therapy Market?

The technological advancements in gene editing, viral vector development, and delivery systems enable the development of more efficient gene-based therapies.

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