Stargardt Disease Clinical and Non-Clinical Studies, Key Companies, Therapeutic Assessment, Emerging Therapies, Treatment Algorithm, and Pipeline Analysis | Belite Bio, Ocugen, Alkeus Pharmaceuticals, Astellas Pharma, SalioGen Therapeutics, Ascidian, Biog

Stargardt Disease Pipeline constitutes 20+ key companies continuously working towards developing 20+ Stargardt Disease treatment therapies, analyzes DelveInsight.

Stargardt Disease Overview:

Stargardt disease is a genetic disorder caused by mutations in both copies of the ABCA4 gene, inherited from each parent. This mutation leads to an abnormal buildup of lipofuscin, a waste material, in the retina. Under normal conditions, the retina efficiently removes this waste, but in individuals with Stargardt disease, this process is impaired, resulting in retinal cell damage and vision loss. The severity of the disease varies widely due to differences in ABCA4 mutations, with some individuals experiencing more significant vision impairment than others. Despite this variability, the underlying mechanism remains the same: defective ABCA4 genes disrupt retinal metabolism, leading to toxic lipofuscin accumulation and progressive retinal degeneration. Ongoing research into these genetic variations may pave the way for targeted treatments and interventions.

The condition primarily affects central vision, causing blurriness, distortion, or dark spots due to macular damage, which is essential for sharp vision. It may also impact color perception and adaptation to light changes.

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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 depicts a robust space with 20+ active players working to develop 20+ pipeline therapies for Stargardt Disease treatment.
Key Stargardt Disease companies such as Belite Bio, Ocugen, Alkeus Pharmaceuticals, Astellas Pharma, SalioGen Therapeutics, Ascidian, Biogen, Splice Bio, and others are evaluating new drugs for Stargardt Disease to improve the treatment landscape.
Promising Stargardt Disease pipeline therapies in various stages of development include OCU410, KIO-301, Tinlarebant, and others.

Recent breakthroughs in the Stargardt Disease Pipeline Segment:
Following a productive 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 due to Stargardt macular degeneration. Site selection is expected to begin in Q4 2024, with patient dosing commencing in Q1 2025
The FDA cleared SpliceBio's IND application for SB-007, a gene therapy targeting the genetic cause of Stargardt disease. Phase 1/2 trials are scheduled to begin in 2025.
In December 2024, ViGeneron GmbH received FDA clearance for its IND application for VG801, a novel mRNA trans-splicing gene therapy designed to treat Stargardt disease and other ABCA4-linked retinal dystrophies. This approval allows for a Phase 1/2 trial to evaluate the therapy's safety, tolerability, and initial efficacy.
In January 2024, the FDA cleared Ascidian Therapeutics' investigational new drug (IND) application and granted Fast Track designation for ACDN-01, marking it as the first clinical-stage RNA exon editor targeting the genetic cause of Stargardt disease. Ascidian plans to initiate enrollment in the Phase 1/2 STELLAR study in 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.

Download our free sample page report on Stargardt Disease pipeline insights @ https://www.delveinsight.com/sample-request/stargardt-disease-stgd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Stargardt Disease Emerging Drugs

Tinlarebant: Belite Bio
KIO-301: Kiora Pharmaceuticals
OCU410: Ocugen

Stargardt Disease Companies

More than 20 key companies are actively developing therapies for Stargardt Disease. Among them, Belite Bio has a drug candidate in 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

Download Sample Pages to Get an in-depth Assessment of the Emerging Stargardt Disease Therapies and Key Companies: Stargardt Disease Clinical Trials and advancements @ https://www.delveinsight.com/report-store/stargardt-disease-stgd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

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 @ https://www.delveinsight.com/sample-request/stargardt-disease-stgd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

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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Contact Info:
Kritika Rehani
krehani@delveinsight.com
info@delveinsight.com
+14699457679

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.

This release was published on openPR.