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.
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Key Takeaways from the Stargardt Disease Market Report
- Among the 7MM, the United States accounted for the highest number of diagnosed prevalent cases of Stargardt, which is 43.0% of the diagnosed-incident cases of Stargardt in 2023.
- In the US, out of all age groups, the highest onset age-specific cases accounted for >20 years, followed by 20-39 years in 2020. In contrast, the least onset age-specific cases were observed in =60 years age groups.
- Among the EU4 and the UK, Germany accounted for the highest number of Stargardt Disease cases, followed by the UK, whereas Spain accounted for the lowest number of prevalent cases.
- In Japan, the highest symptoms-specific cases of Symptomatic Stargardt Disease were for reduced vision, followed by Nyctalopia, Photophobia, and other cases, respectively, in 2023.
- The leading Stargardt Disease Companies such as 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
- Promising Stargardt Disease Therapies such as 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
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Stargardt Disease Epidemiology Segmentation in the 7MM
- Stargardt Disease Diagnosed prevalent cases
- Stargardt Disease Onset-age specific cases
- Stargardt Disease Type-specific cases
- Stargardt Disease Symptom-specific and treated cases
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Emerging Stargardt Disease Therapies
- Emixustat: Kubota Pharmaceuticals
Emixustat, developed by Kubota Pharmaceuticals, is an oral drug initially designed to target the dry form of age-related macular degeneration. It functions by slowing the buildup of toxic waste products that contribute to retinal degeneration in various retinal conditions, including Stargardt disease. The mechanism of action involves the modulation of the visual cycle by inhibiting a critical enzyme in this pathway, Retinal pigment epithelium-specific 65 kDa protein (RPE65). By slowing the visual cycle, Emixustat reduces the availability of vitamin A derivatives (11-cis-and all-trans-retinal) to form precursors of A2E and related compounds. In animal models of Stargardt disease and retinal degeneration, Emixustat has demonstrated the ability to decrease the accumulation of A2E and protect the retina from light-induced damage.
- MCO-010: Nanoscope Therapeutics
Nanoscope’s MCO-010 is an optogenetic gene therapy that utilizes a convenient and well-established intraocular injection to deliver a gene encoding the ambient light-sensitive MCO protein into retinal cells. Multi-Characteristic Opsin (MCO) re-sensitize the retina for detecting low light levels to restore vision in blind patients, specifically for the treatment of Stargardt disease, with the goal of improving visual function.
Stargardt Disease Market Outlook
Stargardt disease is a rare genetic eye disease that occurs when fatty material builds up on the macula, the small part of the retina responsible for sharp, central vision. It is caused by a mutation in the ABCA4 gene (also caused by other genes in some instances), which prevents the production of a protein that cleans up the fatty material left over from vitamin A metabolism. As a result, the fatty material accumulates in yellowish clumps on the macula, eventually killing the light-sensitive cells and destroying central vision. Many new molecules with novel mechanisms, like emixustat, gildeuretinol, tinlarebant, avacincaptad pegol, among others, and gene therapies like MCO-010 and others, are being developed for the treatment of Stargardt disease by key players like Kubota Vision, Nanoscope Therapeutics, Alkeus Pharmaceuticals, Belite Bio, Astellas Pharma among others.
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Stargardt Disease Therapies and 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
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
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Scope of the Stargardt Disease Market Report
- Study Period: 2020–2034
- Coverage: 7MM
- 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
- 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
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
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