DelveInsight’s, “Stargardt Disease Pipeline Insight, 2024,” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Stargardt Disease (STGD) 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.

 

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Key Takeaways from the Stargardt Disease Pipeline Report

• In August 2024:- Belite Bio Inc.- A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease.
• DelveInsight’s Stargardt Disease pipeline report depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Stargardt Disease treatment.
• The leading Stargardt Disease Companies such as Belite Bio, Ocugen, Alkeus Pharmaceuticals, Astellas Pharma, SalioGen Therapeutics, Ascidian, Biogen, Splice Bio, and others.
• Promising Stargardt Disease Therapies such as OCU410ST, Tinlarebant, ALK-001, ACDN-01, avacincaptad pegol, and others.

 

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Stargardt Disease Emerging Drugs Profile

• Tinlarebant: Belite Bio

Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1 and also contribute to disease progression in GA, or advanced Dry AMD. Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the US, and Orphan Drug Designation in the U.S., Europe, and Japan for the treatment of STGD1. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Stargardt disease.

• KIO-301: Kiora Pharmaceuticals

KIO-301 is a molecular photoswitch designed to confer light-sensing capabilities to Retinal Ganglion Cells (RGCs). It targets patients with inherited retinal diseases where mutations lead to photoreceptor death, causing vision loss. Unlike healthy eyes where photoreceptors perform light detection, KIO-301 selectively enters RGCs downstream of degenerated photoreceptors. Inside RGCs, it interacts with voltage-gated ion channels. When exposed to light, KIO-301 changes shape, altering current flow and activating neurons to signal the brain. Removal of light causes KIO-301 to revert to its original shape, halting signaling. Essentially, KIO-301 acts as a light switch within the eye, restoring light sensitivity in patients with retinal degeneration. Currently, the drug is in Phase II stage of its clinical trial for the treatment of stargardt disease.

• OCU410: Ocugen

OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA. Currently, the drug is in Phase I/II stage of its clinical trial for the treatment of stargardt disease.

 

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Stargardt disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration

• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

 

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

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

 

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Scope of the Stargardt Disease Pipeline Report

• Coverage- Global
• Stargardt Disease Companies- Belite Bio, Ocugen, Alkeus Pharmaceuticals, Astellas Pharma, SalioGen Therapeutics, Ascidian, Biogen, Splice Bio, and others.
• Stargardt Disease Therapies- OCU410ST, Tinlarebant, ALK-001, ACDN-01, avacincaptad pegol, and others.
• Stargardt Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Stargardt Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

 

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Table of Content

1. Introduction
2. Executive Summary
3. Stargardt Disease: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Stargardt disease – DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Tinlarebant: Belite Bio
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. KIO-301: Kiora Pharmaceuticals
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. OCU410: Ocugen
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug name: Company name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Stargardt disease Key Companies
21. Stargardt disease Key Products
22. Stargardt disease -Unmet Needs
23. Stargardt disease -Market Drivers and Barriers
24. Stargardt disease -Future Perspectives and Conclusion
25. Stargardt disease Analyst Views
26. Stargardt disease Key Companies
27. Appendix

 

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