Stargardt Disease Pipeline

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.

Geography Covered

• Global coverage

Stargardt Disease: Understanding

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 the 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 the 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, a detailed description of the drug is given which includes the 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.

Stargardt Disease Emerging Drugs Chapters

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 

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.

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 the following parameters that define the scope of the report, such as:

Major  Players in Stargardt Disease 

There are approx. 20+ key companies that are developing therapies for Stargardt Disease. The companies that have their Stargardt Disease drug candidates in the most advanced stage, i.e. Phase III include Belite Bio.

Phases

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

Route of Administration

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

Molecule Type

Products have been categorized under various Molecule types such as

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

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

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 detailed information on collaborations, acquisition and merger, and 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 

Stargardt Disease Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

• 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, and licensing activities related to the Stargardt Disease therapeutics? 
• What are the recent trends, drug types and novel technologies developed to overcome the limitations 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?

Key Players

• Belite Bio
• Ocugen
• Alkeus Pharmaceuticals
• Astellas Pharma
• SalioGen Therapeutics
• Ascidian 
• Biogen
• Splice Bio

Key Products

• Tinlarebant
• OCU410ST
• ALK-001
• ASP-7317
• SGT-1001
• ABCA4 
• NSR-ABCA4
• SB-007