The Stargardt Disease market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Stargardt Disease pipeline products will significantly revolutionize the Stargardt Disease market dynamics.

DelveInsight’s “Stargardt Disease Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Stargardt Disease, historical and forecasted epidemiology as well as the Stargardt Disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Stargardt Disease market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

To Know in detail about the Stargardt Disease market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Stargardt Disease Market Insights

Some of the key facts of the Stargardt Disease Market Report:

• The Stargardt Disease market size was valued ~USD 27 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034).

• In January 2026, Ocugen, Inc. reported encouraging outcomes from its Phase 1 GARDian1 study of OCU410ST, an innovative gene therapy being developed for Stargardt disease, published in Nature Eye. The study showed a favorable safety and tolerability profile, along with meaningful clinical benefits over a 12-month period. Notably, treated patients experienced a 54% reduction in atrophic lesion progression and improvements in visual acuity. Unlike mutation-specific approaches, OCU410ST is designed to alter the underlying disease course, positioning it as a potentially transformative option for the more than 100,000 individuals affected across the U.S. and Europe, where no approved therapies currently exist. Meanwhile, the Phase 2/3 GARDian3 trial is progressing ahead of plan, with a Biologics License Application (BLA) submission targeted for 2027.

• In December 2025, A Phase 3 study evaluating Belite Bio’s tinlarebant in adolescents with a rare inherited eye disorder has achieved its primary endpoint, prompting the company to include the U.S. among the countries where it plans to seek approval next year. The trial enrolled 104 participants with Stargardt disease type 1 (STGD1), who were assigned to either tinlarebant or placebo. STGD1 leads to lesion formation in the retina and progressive central vision loss. Tinlarebant, an oral therapy, aims to limit the buildup of vitamin A–derived toxins that fuel the disease. The treatment produced a 35.7% reduction in lesion growth compared to placebo. A post hoc analysis accounting for disease progression showed similar outcomes, as did additional lesion-growth metrics.

• In September 2025, VeonGen Therapeutics was granted FDA RMAT designation for VG801, a gene therapy designed to treat Stargardt disease and other ABCA4-related retinal disorders. VG801 employs a dual AAV vector along with the company’s vgRNA REVeRT platform to deliver a functional ABCA4 gene using an engineered vgAAV capsid targeted to photoreceptor cells.

• In August 2025, AAVantgarde Bio was granted FDA Fast Track Designation for AAVB-039, its advanced gene therapy targeting Stargardt disease caused by biallelic ABCA4 mutations. Additionally, the FDA has approved the IND application for AAVB-039, allowing the therapy to progress into clinical development.

• In June 2025, Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company at the forefront of gene therapies for vision-related diseases, has announced that the U.S. Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) amendment to launch a Phase 2/3 pivotal confirmatory trial of OCU410ST. This modifier gene therapy candidate is being developed to treat all forms of Stargardt disease (ABCA4-associated retinopathies). OCU410ST has already received both Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA for the treatment of ABCA4-related conditions, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

• In March 2025, Barcelona-based genetic medicines company, SpliceBio, has administered the first dose in the Phase 1/2 ASTRA clinical trial (NCT identifier pending) evaluating SB-007, a dual adeno-associated virus (AAV) vector-based gene therapy for Stargardt disease. This inherited retinal disorder, caused by mutations in the ABCA4 gene, leads to progressive vision loss and currently has no approved treatments. Affecting approximately 1 in 8,000 to 10,000 people, it is the most common form of inherited juvenile macular degeneration.

• In February 2025, Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company specializing in gene therapies for blindness diseases, has secured alignment with the U.S. Food and Drug Administration (FDA) to proceed with a Phase 2/3 pivotal confirmatory trial for OCU410ST. If the trial yields positive results, it could serve as the foundation for a biologics license application (BLA) submission.

• In January 2025, Alkeus Pharmaceuticals, Inc., a biopharmaceutical company focused on retinal disease treatment, has reported positive interim results from its TEASE-3 study. The data indicate that early-stage Stargardt disease patients receiving oral gildeuretinol acetate exhibited no disease progression and maintained stable visual acuity over multiple years.

• In September 2024, Nanoscope Therapeutics Inc., a clinical-stage biotechnology firm focused on gene therapies for retinal degenerative diseases and age-related macular degeneration (AMD), announced a successful End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA). The meeting focused on the clinical program assessing MCO-010 for treating severe vision loss caused by Stargardt Macular Degeneration (SMD), paving the way for the progression of MCO-010 to a Phase 3 registrational trial.

• According to DelveInsight’s estimates, the United States held the largest share of the Stargardt Disease treatment market in 2023, representing nearly 55% of the total market value, surpassing the combined market share of the EU4, the UK, and Japan.

• By 2034, MCO-010 (Sonpiretigene Isteparvovec) is projected to hold the largest market share across the 7MM, with ALK-001 (gildeuretinol) expected to follow as the second-leading therapy.

• During the 2024–2034 forecast period, the Stargardt Disease market size is projected to grow significantly, driven by emerging pipeline therapies such as ALK-001 (gildeuretinol), Tinlarebant (LSB-008), IZERVAY (avacincaptad pegol), Emixustat, along with gene therapy candidates like MCO-010 (Sonpiretigene Isteparvovec) and others. These advancing treatments are expected to play a key role in market expansion.

• Stargardt disease, also called Stargardt’s macular dystrophy or juvenile macular degeneration, is a rare inherited eye disorder caused by the accumulation of fatty deposits on the macula—the central part of the retina responsible for detailed vision. It is the most common form of recessive macular dystrophy in children and is estimated to affect about 10 to 12.5 individuals per 100,000 in the United States.

• Across the 7MM regions, the United States recorded the largest share of diagnosed prevalent cases of Stargardt Disease in 2023, representing 43% of all diagnosed incidents.

• In the US, the greatest number of age-specific onset cases in 2020 occurred in individuals aged over 20 years, followed by those between 20 and 39 years. In comparison, the lowest number of cases was reported in people aged 60 years and above.

• Key Stargardt Disease Companies: Ocugen, Kubota Vision Inc, Nanoscope Therapeutics Inc., RBP4 Pty Ltd, Stargazer Pharmaceuticals, Inc., Belite Bio, Inc, Alkeus Pharmaceuticals, Inc., IVERIC bio, Inc., Kubota Vision Inc., Astellas Institute, Nanoscope Therapeutics, Alkeus Pharmaceuticals, and others

• Key Stargardt Disease Therapies: OCU410ST, Emixustat, Gene Therapy-vMCO-010, tinlarebant, STG-001, Tinlarebant, ALK-001, Zimura, Emixustat, MA09-hRPE, MCO-010 (Sonpiretigene Isteparvovec), ALK-001 (Gildeuretinol), and others

• The Stargardt Disease market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Stargardt Disease pipeline products will significantly revolutionize the Stargardt Disease market dynamics.

Stargardt Disease Overview

Stargardt disease is a rare inherited eye disorder that causes progressive vision loss, primarily affecting the macula the part of the retina responsible for sharp, central vision. It is most commonly caused by mutations in the ABCA4 gene, leading to the buildup of toxic fatty deposits in retinal cells. Symptoms typically begin in childhood or adolescence and may include blurred vision, difficulty seeing in low light, and loss of color perception. Over time, the condition can lead to significant central vision impairment, though peripheral vision is usually preserved. There is currently no cure, but several emerging therapies and gene treatments are under development.

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Stargardt Disease Market

The dynamics of the Stargardt Disease market are anticipated to change in the coming years owing to the expected launch of emerging therapies and others during the forecasted period 2020-2034.

“In the near future, there are some positive candidates in the developmental stage by key players such as Acucela Inc (Kubota), Alkeus Pharmaceuticals Inc, Astellas Pharma Inc, Revision Therapeutics and others.”

Stargardt Disease 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.

Stargardt Disease Epidemiology Segmentation:

The Stargardt Disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Prevalence of Stargardt Disease

• Prevalent Cases of Stargardt Disease by severity

• Gender-specific Prevalence of Stargardt Disease

• Diagnosed Cases of Episodic and Chronic Stargardt Disease

Download the report to understand which factors are driving Stargardt Disease epidemiology trends @ Stargardt Disease Epidemiological Insights

Stargardt Disease Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Stargardt Disease market or expected to get launched during the study period. The analysis covers Stargardt Disease market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Stargardt Disease Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Stargardt Disease Therapies and Key Companies

• OCU410ST: Ocugen

• Emixustat: Kubota Vision Inc

• Gene Therapy-vMCO-010: Nanoscope Therapeutics Inc.

• tinlarebant: RBP4 Pty Ltd

• STG-001: Stargazer Pharmaceuticals, Inc.

• Tinlarebant: Belite Bio, Inc

• ALK-001: Alkeus Pharmaceuticals, Inc.

• Zimura: IVERIC bio, Inc.

• Emixustat: Kubota Vision Inc.

• MA09-hRPE: Astellas Institute

• Emixustat : Kubota Pharmaceuticals

• MCO-010 (Sonpiretigene Isteparvovec): Nanoscope Therapeutics

• ALK-001 (Gildeuretinol): Alkeus Pharmaceuticals

To know more about Stargardt Disease treatment, visit @ Stargardt Disease Medications

Stargardt Disease Market Strengths

• Stargardt Disease has a well-identified genetic basis, primarily associated with mutations in the ABCA4 gene, allowing for targeted research and potential therapeutic interventions.

• Regulatory agencies, such as the FDA, recognize Stargardt Disease as an orphan disease, providing incentives for the development of orphan drugs and accelerated approval pathways.

Stargardt Disease Market Opportunities

• Ongoing research in gene therapies, pharmacological interventions, and emerging technologies provides opportunities for the development of novel and effective treatments.

• Rapid advancements in genetic technologies, including CRISPR-Cas9, offer potential breakthroughs in gene editing and correction of ABCA4 mutations

Scope of the Stargardt Disease Market Report

• Study Period: 2020–2034

• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

• Key Stargardt Disease Companies: Ocugen, Kubota Vision Inc, Nanoscope Therapeutics Inc., RBP4 Pty Ltd, Stargazer Pharmaceuticals, Inc., Belite Bio, Inc, Alkeus Pharmaceuticals, Inc., IVERIC bio, Inc., Kubota Vision Inc., Astellas Institute, Nanoscope Therapeutics, Alkeus Pharmaceuticals, and others

• Key Stargardt Disease Therapies: OCU410ST, Emixustat, Gene Therapy-vMCO-010, tinlarebant, STG-001, Tinlarebant, ALK-001, Zimura, Emixustat, MA09-hRPE, MCO-010 (Sonpiretigene Isteparvovec), ALK-001 (Gildeuretinol), Zimura, and others

• Stargardt Disease Therapeutic Assessment: Stargardt Disease current marketed and Stargardt Disease emerging therapies

• Stargardt Disease Market Dynamics: Stargardt Disease market drivers and Stargardt Disease market barriers

• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies

• Stargardt Disease Unmet Needs, KOL’s views, Analyst’s views, Stargardt Disease Market Access and Reimbursement

Discover more about therapies set to grab major Stargardt Disease market share @ Stargardt Disease Treatment Landscape

Table of Contents

1. Stargardt Disease Market Report Introduction

2. Executive Summary for Stargardt Disease

3. SWOT analysis of Stargardt Disease

4. Stargardt Disease Patient Share (%) Overview at a Glance

5. Stargardt Disease Market Overview at a Glance

6. Stargardt Disease Disease Background and Overview

7. Stargardt Disease Epidemiology and Patient Population

8. Country-Specific Patient Population of Stargardt Disease

9. Stargardt Disease Current Treatment and Medical Practices

10. Stargardt Disease Unmet Needs

11. Stargardt Disease Emerging Therapies

12. Stargardt Disease Market Outlook

13. Country-Wise Stargardt Disease Market Analysis (2020–2034)

14. Stargardt Disease Market Access and Reimbursement of Therapies

15. Stargardt Disease Market Drivers

16. Stargardt Disease Market Barriers

17. Stargardt Disease Appendix

18. Stargardt Disease Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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