Stargardt Disease Clinical Trials, Companies, Therapeutic Assessment, Therapies, Treatment Algorithm, Pipeline Belite Bio, Ocugen, Alkeus Pharmaceuticals, Astellas Pharma, Saliogen Therapeutics, Asc

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"Stargardt Disease Clinical Trials"DelveInsight's,“Stargardt Disease - Pipeline Insight, 2025” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in the 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.

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

• 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.

• 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.

• 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.

• 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.

• 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.

• 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:

• The report provides detailed insights into the key companies that are developing therapies in the Stargardt Disease Market.

• The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Stargardt Disease treatment.

• It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• 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.

• 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

• Tinlarebant: Belite Bio

• KIO-301: Kiora Pharmaceuticals

• 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

• 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

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

• Intravenous

• Subcutaneous

• Oral

• Intramuscular

Stargardt Disease Products have been categorized under various Molecule types such as

• Monoclonal antibody

• Small molecule

• 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

Download Stargardt Disease Sample report to know in detail about the Stargardt Disease treatment market @ Stargardt Disease Therapeutic Assessment

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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