According to DelveInsight, the Stargardt Disease pipeline features over 20 leading companies actively developing more than 20 therapeutic candidates for the treatment of Stargardt Disease.
Stargardt Disease Overview:
Stargardt disease is an inherited genetic disorder caused by mutations in both copies of the ABCA4 gene, one from each parent. These mutations result in the abnormal accumulation of lipofuscin, a cellular waste product, in the retina. Normally, the retina can efficiently clear this waste, but in Stargardt disease, this process is impaired, leading to retinal cell damage and progressive vision loss. The severity of the condition varies depending on the specific ABCA4 mutations, with some individuals experiencing more pronounced vision impairment than others. Regardless of severity, the core mechanism involves defective ABCA4 genes disrupting retinal metabolism, causing toxic lipofuscin buildup and gradual retinal degeneration. Research into these genetic variations is ongoing and may enable the development of targeted therapies.
Stargardt disease primarily affects central vision, leading to blurriness, visual distortion, or dark spots due to damage to the macula, which is critical for sharp vision. It can also impair color perception and the ability to adapt to changes in lighting.
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"Stargardt Disease Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Stargardt Disease Therapeutics Market.
Key Takeaways from the Stargardt Disease Pipeline Report
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DelveInsight’s Stargardt Disease pipeline report highlights an active and evolving landscape, with over 20 companies developing more than 20 therapeutic candidates for the treatment of Stargardt Disease.
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Leading companies—including Belite Bio, Ocugen, Alkeus Pharmaceuticals, Astellas Pharma, SalioGen Therapeutics, Ascidian, Biogen, Splice Bio, and others—are actively exploring new drugs to enhance the Stargardt Disease treatment landscape. Promising pipeline candidates in various stages of development include OCU410, KIO-301, Tinlarebant, and others.
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Following a successful End-of-Phase 2 meeting with the FDA, Nanoscope Therapeutics plans to initiate a Phase 3 clinical trial for its optogenetic therapy, MCO-010, aimed at treating severe vision loss from Stargardt macular degeneration. Site selection is expected in Q4 2024, with patient dosing beginning in Q1 2025.
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The FDA cleared SpliceBio’s IND application for SB-007, a gene therapy targeting the genetic cause of Stargardt disease, with Phase 1/2 trials scheduled to start in 2025.
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In December 2024, ViGeneron GmbH received FDA IND clearance for VG801, a novel mRNA trans-splicing gene therapy designed to treat Stargardt disease and other ABCA4-linked retinal dystrophies, enabling a Phase 1/2 trial to evaluate safety, tolerability, and initial efficacy.
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In January 2024, the FDA cleared Ascidian Therapeutics’ IND application and granted Fast Track designation for ACDN-01, the first clinical-stage RNA exon editor targeting the genetic cause of Stargardt disease. Enrollment for the Phase 1/2 STELLAR study is planned for the first half of 2024.
Stargardt Disease Pipeline Analysis
The report provides insights into:
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The report provides detailed insights into the key companies that are developing therapies in the Stargardt Disease Market.
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The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Stargardt Disease treatment.
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It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Stargardt Disease market.
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Stargardt Disease Emerging Drugs
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Tinlarebant: Belite Bio
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KIO-301: Kiora Pharmaceuticals
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OCU410: Ocugen
Stargardt Disease Companies
Over 20 leading companies are actively working on therapies for Stargardt Disease, with Belite Bio having a drug candidate that has reached the most advanced stage of development, Phase III.
DelveInsight’s report covers around 20+ products under different phases of clinical development like
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Late stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I) along with the details of
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
Stargardt Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
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Intravenous
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Subcutaneous
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Oral
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Intramuscular
Stargardt Disease Products have been categorized under various Molecule types such as
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Monoclonal antibody
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Small molecule
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Peptide
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Stargardt Disease Pipeline Therapeutic Assessment
• Stargardt Disease Assessment by Product Type
• Stargardt Disease By Stage
• Stargardt Disease Assessment by Route of Administration
• Stargardt Disease Assessment by Molecule Type
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Table of Content
1. Report Introduction
2. Executive Summary
3. Stargardt Disease Current Treatment Patterns
4. Stargardt Disease - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Stargardt Disease Late-Stage Products (Phase-III)
7. Stargardt Disease Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Stargardt Disease Discontinued Products
13. Stargardt Disease Product Profiles
14. Stargardt Disease Key Companies
15. Stargardt Disease Key Products
16. Dormant and Discontinued Products
17. Stargardt Disease Unmet Needs
18. Stargardt Disease Future Perspectives
19. Stargardt Disease Analyst Review
20. Appendix
21. Report Methodology
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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.
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