Stargardt disease (STGD1) is the most common form of juvenile macular degeneration, characterized by progressive central vision loss caused by mutations in the ABCA4 gene. According to Jeroen A. A. H. Pas et al. (2024), its point prevalence is approximately 1:22,000–19,000, often leading to severe visual impairment or blindness. Current therapeutic approaches in development include gene therapy, small-molecule visual cycle modulators, anti-lipofuscin agents, and cell-based regenerative therapies. According to the Stargardt disease pipeline analysis by Expert Market Research, growing R&D focus, improved diagnostics, and increasing diagnosed patient populations are expected to drive significant market growth in the coming years.
Major companies involved in the Stargardt disease pipeline analysis include Ocugen, Splice Bio, and others.
Leading drugs currently in the pipeline include OCU410ST, ALK-001, SB-007, and others.
The pipeline is poised for substantial expansion, driven by advancements in gene therapies and novel pharmacological candidates. Several programs are advancing through late-stage development, highlighting strong potential for innovative treatment options and market growth.
The Stargardt Disease Pipeline Analysis Report by Expert Market Research gives comprehensive insights into Stargardt disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Stargardt disease. The Stargardt disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The Stargardt disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with Stargardt disease treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to Stargardt disease.
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Stargardt disease is a hereditary retinal disorder caused by mutations in the ABCA4 gene, resulting in the malfunction of photoreceptor cells in the retina. The condition primarily causes progressive central vision loss, often beginning in childhood or adolescence, which gradually impairs visual acuity, affects daily activities such as reading and recognizing faces, and can ultimately lead to legal blindness if left unmanaged.
Stargardt disease treatments focus on experimental approaches, including gene therapies, stem cell therapies, and pharmacological interventions, aiming to slow progression, restore retinal function, or correct underlying genetic defects. In August 2025, VeonGen Therapeutics’ VG801 gene therapy received the FDA Regenerative Medicine Advanced Therapy designation for Stargardt disease. VG801 delivers a functional ABCA4 gene, addressing the root genetic cause, and is currently in Phase I/II clinical trials, representing a promising advancement in the Stargardt disease drug pipeline.
According to Jeroen A. A. H. Pas et al., 2024, Stargardt disease (STGD1) is the most common form of juvenile macular degeneration, with a point prevalence of 1 in 19,000–22,000, often leading to severe visual impairment or blindness. As per the American Academy of Ophthalmology, the disease affects over 30,000 people in the United States and around 150,000 worldwide. According to Cornish et al., the annual incidence in the United Kingdom ranges from 0.11 to 0.12 per 100,000. A Dutch study reports 1.67–1.95 cases per 1,000,000 annually, emphasizing the rarity and clinical significance of STGD1.
This section of the report covers the analysis of Stargardt disease drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
By Drug Class
The Stargardt disease pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration.
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II, with 52%, covers a major share of the total Stargardt disease clinical trials. It is followed by phase I at 33% and Phase III, (accounting for 9%). These positive distributions highlight a robust pipeline, indicating significant market growth potential, faster drug approvals, and improved treatment options for patients.
The drug molecule categories covered under the Stargardt disease pipeline analysis include small molecules, monoclonal antibodies, gene therapies, peptides, and polymers. The Stargardt disease report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Stargardt disease. Gene therapies are emerging as a promising approach for treating Stargardt disease, the most common inherited form of macular degeneration caused by ABCA4 gene mutations. AAVB-039, an adeno-associated virus-based therapy, targets the underlying genetic defect by delivering the full-length ABCA4 protein to retinal cells, aiming to slow or prevent vision loss. In October 2025, it received FDA Orphan Drug Designation, recognizing its potential for this rare retinal disorder. AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial, assessing safety, tolerability, and preliminary efficacy in patients with Stargardt disease.
The EMR report for the Stargardt disease pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed Stargardt disease therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in Stargardt disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Stargardt disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Stargardt disease drug candidates.
OCU410ST is a gene therapy drug sponsored by Ocugen, designed for the treatment of Stargardt disease. This Phase 2/3 pivotal confirmatory trial is enrolling fifty-one subjects in a randomized, outcome assessor-masked, multicenter study to examine the safety and efficacy of a single subretinal injection of OCU410ST. The drug uses an Adeno-Associated Virus serotype 5 (AAV5) to deliver the human RORA gene, targeting pathways involved in lipid metabolism, oxidative stress, inflammation, and cell survival, aiming to restore retinal homeostasis.
ALK-001, sponsored by Alkeus Pharmaceuticals, Inc., is an oral gildeuretinol acetate designed to reduce the dimerization of vitamin A without affecting the visual cycle. This Phase 2 open-label extension study is examining the long-term safety, tolerability, pharmacokinetics, and effects of ALK-001 on Stargardt disease progression in participants aged 8 and above. ALK-001 has shown preclinical efficacy in preventing retinal degeneration and preserving visual function, while prior clinical trials demonstrated significant slowing of retinal lesion growth. The study is continuing to evaluate its therapeutic potential and safety profile.
SB-007 is an investigational gene therapy sponsored by Splice Bio, designed to treat Stargardt disease (STGD1) by restoring functional ABCA4 protein in photoreceptor cells. This Phase 1/2 study is examining the safety, tolerability, and preliminary efficacy of a single subretinal injection of SB-007 in approximately 57 participants over 96 weeks. SB-007 uses dual AAV8 vectors and proprietary protein splicing (split-inteins) to reconstitute the full-length ABCA4 protein, potentially slowing vision loss. The study is actively recruiting and aims to determine safe dosing and early efficacy signals for STGD1 patients.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Stargardt Disease Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Stargardt disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into Stargardt disease collaborations, regulatory environments, and potential growth opportunities.
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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