Stargardt Disease Pipeline Drugs Analysis Report 2025: Latest Innovations, Promising Drugs, and Clinical Advancements in Ophthalmology

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 Stargardt Disease 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

* On 27 August 2025, Belite Bio Inc. initiated a study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.
* 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 Emixustat, OCU410ST, tinlarebant, SB-007, STG-001, ALK-001, Emixustat, ACDN-01, VG801 and others.

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The Stargardt Disease Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Stargardt Disease Pipeline Report also highlights the unmet needs with respect to the Stargardt Disease.

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.

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 US, Europe, and Japan for the treatment of STGD1. Currently, the drug is in the 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 the 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 the Phase I/II stage of its clinical trial for the treatment of stargardt disease.

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Stargardt Disease Companies

Belite Bio, Ocugen, Alkeus Pharmaceuticals, Astellas Pharma, SalioGen Therapeutics, Ascidian, Biogen, Splice Bio and others.

The Stargardt Disease Pipeline report provides insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Stargardt Disease with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Stargardt Disease Treatment.
* Stargardt Disease Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Stargardt Disease 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.

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

* Oral
* Intravenous
* Subcutaneous
* Parenteral
* Topical

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

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

From emerging drug candidates to competitive intelligence, the Stargardt Disease Pipeline Report covers it all - check it out now @ Stargardt Disease Market Drivers and Barriers, and Future Perspectives [https://www.delveinsight.com/sample-request/stargardt-disease-stgd-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

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- Emixustat, OCU410ST, tinlarebant, SB-007, STG-001, ALK-001, Emixustat, ACDN-01, VG801 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 Contents

* Introduction
* Executive Summary
* Stargardt disease: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Stargardt disease - DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* Tinlarebant: Belite Bio
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* KIO-301: Kiora Pharmaceuticals
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* OCU410: Ocugen
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* Drug Name: Company Name
* Drug profiles in the detailed report.....
* Inactive Products
* 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

About Us

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