
Stargardt Disease - Pipeline Insight, 2025
Description
DelveInsight’s, “Stargardt Disease - Pipeline Insight, 2025” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Stargardt Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Stargardt Disease: Overview
Stargardt disease, stemming from a hereditary anomaly in the ABCA4 gene, manifests when both copies of this gene, one from each parent, carry mutations. This genetic defect leads to the accumulation of lipofuscin, a metabolic waste product, within the retina. Normally, the retina efficiently clears away this material, but in Stargardt disease, this clearance process is hindered, resulting in damage to retinal cells and subsequent vision loss. The severity of the condition varies widely among individuals due to the diverse spectrum of mutations within the ABCA4 gene. Consequently, some individuals experience more profound vision impairment than others. Despite the variability in manifestation, the core mechanism remains consistent: defective ABCA4 genes disrupt the delicate balance of retinal metabolism, leading to the detrimental accumulation of lipofuscin and subsequent degeneration of retinal cells. As research progresses, understanding these genetic variations holds promise for tailored treatments and interventions for individuals affected by Stargardt disease.
Stargardt disease presents a range of symptoms, primarily affecting central vision with manifestations such as blurry, distorted, or dark areas, owing to damage in the macula, critical for detailed central vision. Color perception and light adjustment may also be compromised.
Diagnosis typically involves a thorough eye examination to detect characteristic yellowish lipofuscin deposits in and around the macula, complemented by tests like fluorescein angiography and genetic analysis to pinpoint the specific genetic mutation. Regrettably, a definitive cure for Stargardt disease remains elusive, though ongoing research explores various avenues such as gene therapy, nanoparticle interventions, anti-sense oligonucleotides, pharmaceutical approaches, dietary supplements, and cell replacement therapy. While reversal of Stargardt disease isn't currently feasible, strategies like vision rehabilitation and the use of low vision aids empower individuals to manage symptoms effectively and maintain engagement in daily activities despite vision impairment. As research advances, these therapeutic options offer hope for enhancing the quality of life for those affected by Stargardt disease.
""Stargardt Disease - Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Stargardt Disease pipeline landscape is provided which includes the disease overview and Stargardt Disease treatment guidelines. The assessment part of the report embraces, in depth Stargardt Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Stargardt Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Stargardt Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Stargardt Disease Emerging Drugs
Further product details are provided in the report…….
Stargardt Disease: Therapeutic Assessment
This segment of the report provides insights about the different Stargardt Disease drugs segregated based on following parameters that define the scope of the report, such as:
Stargardt Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Stargardt Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Stargardt Disease drugs.
Stargardt Disease Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Stargardt Disease: Overview
Stargardt disease, stemming from a hereditary anomaly in the ABCA4 gene, manifests when both copies of this gene, one from each parent, carry mutations. This genetic defect leads to the accumulation of lipofuscin, a metabolic waste product, within the retina. Normally, the retina efficiently clears away this material, but in Stargardt disease, this clearance process is hindered, resulting in damage to retinal cells and subsequent vision loss. The severity of the condition varies widely among individuals due to the diverse spectrum of mutations within the ABCA4 gene. Consequently, some individuals experience more profound vision impairment than others. Despite the variability in manifestation, the core mechanism remains consistent: defective ABCA4 genes disrupt the delicate balance of retinal metabolism, leading to the detrimental accumulation of lipofuscin and subsequent degeneration of retinal cells. As research progresses, understanding these genetic variations holds promise for tailored treatments and interventions for individuals affected by Stargardt disease.
Stargardt disease presents a range of symptoms, primarily affecting central vision with manifestations such as blurry, distorted, or dark areas, owing to damage in the macula, critical for detailed central vision. Color perception and light adjustment may also be compromised.
Diagnosis typically involves a thorough eye examination to detect characteristic yellowish lipofuscin deposits in and around the macula, complemented by tests like fluorescein angiography and genetic analysis to pinpoint the specific genetic mutation. Regrettably, a definitive cure for Stargardt disease remains elusive, though ongoing research explores various avenues such as gene therapy, nanoparticle interventions, anti-sense oligonucleotides, pharmaceutical approaches, dietary supplements, and cell replacement therapy. While reversal of Stargardt disease isn't currently feasible, strategies like vision rehabilitation and the use of low vision aids empower individuals to manage symptoms effectively and maintain engagement in daily activities despite vision impairment. As research advances, these therapeutic options offer hope for enhancing the quality of life for those affected by Stargardt disease.
""Stargardt Disease - Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Stargardt Disease pipeline landscape is provided which includes the disease overview and Stargardt Disease treatment guidelines. The assessment part of the report embraces, in depth Stargardt Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Stargardt Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Stargardt Disease R&D. The therapies under development are focused on novel approaches to treat/improve Stargardt Disease.
This segment of the Stargardt Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Stargardt Disease Emerging Drugs
- Tinlarebant: Belite Bio
- KIO-301: Kiora Pharmaceuticals
- OCU410: Ocugen
Further product details are provided in the report…….
Stargardt Disease: Therapeutic Assessment
This segment of the report provides insights about the different Stargardt Disease drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Stargardt Disease
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
- Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
- Product Type
Stargardt Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Stargardt Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Stargardt Disease drugs.
Stargardt Disease Report Insights
- Stargardt Disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Stargardt Disease drugs?
- How many Stargardt Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Stargardt Disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Stargardt Disease therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Stargardt Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
- Belite Bio
- Ocugen
- Alkeus Pharmaceuticals
- Astellas Pharma
- SalioGen Therapeutics
- Ascidian
- Biogen
- Splice Bio
- Tinlarebant
- OCU410ST
- ALK-001
- ASP-7317
- SGT-1001
- ABCA4
- NSR-ABCA4
- SB-007
Table of Contents
80 Pages
- Introduction
- Executive Summary
- Stargardt Disease : Overview
- Introduction
- Causes
- Pathophysiology
- Signs and Symptoms
- Diagnosis
- Treatment
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Stargardt Disease – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Tinlarebant: Belite Bio
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- KIO-301: Kiora Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- OCU410: Ocugen
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Stargardt Disease Key Companies
- Stargardt Disease Key Products
- Stargardt Disease - Unmet Needs
- Stargardt Disease - Market Drivers and Barriers
- Stargardt Disease - Future Perspectives and Conclusion
- Stargardt Disease Analyst Views
- Stargardt Disease Key Companies
- Appendix
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