The Stargardt disease therapeutic landscape is experiencing unprecedented momentum, with multiple gene therapies advancing through clinical development and showing promising results for this rare inherited retinal disorder that affects approximately 1 in 8,000 to 10,000 people worldwide.
Gene Therapy Breakthroughs Lead Clinical Development
Ocugen, Inc. (NASDAQ: OCGN) achieved a major regulatory milestone in June 2025 when the U.S. Food and Drug Administration (FDA) approved the Investigational New Drug (IND) amendment to launch a Phase 2/3 pivotal confirmatory trial of OCU410ST. This modifier gene therapy candidate is being developed to treat all forms of Stargardt disease (ABCA4-associated retinopathies) and has already received both Rare Pediatric Disease Designation and Orphan Drug Designation from the FDA for the treatment of ABCA4-related conditions, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.
The company secured alignment with the FDA in February 2025 to proceed with the Phase 2/3 pivotal confirmatory trial for OCU410ST. If the trial yields positive results, it could serve as the foundation for a biologics license application (BLA) submission. OCU410ST utilizes an adeno-associated virus (AAV) delivery system to administer the RAR Related Orphan Receptor A (RORA) gene to the retina.
Barcelona-based genetic medicines company SpliceBio marked another significant advancement in March 2025 by administering the first dose in the Phase 1/2 ASTRA clinical trial evaluating SB-007, a dual adeno-associated virus (AAV) vector-based gene therapy for Stargardt disease. This inherited retinal disorder, caused by mutations in the ABCA4 gene, leads to progressive vision loss and currently has no approved treatments.
Oral Therapy Shows Disease Stabilization
Alkeus Pharmaceuticals, Inc., a biopharmaceutical company focused on retinal disease treatment, reported positive interim results from its TEASE-3 study in January 2025. The data indicate that early-stage Stargardt disease patients receiving oral gildeuretinol acetate exhibited no disease progression and maintained stable visual acuity over multiple years. The company revealed in May 2024 that additional favorable findings from the TEASE-3 clinical trial investigating gildeuretinol in adolescent and young-adult patients with early-stage Stargardt disease would be disclosed at the Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting.
Pipeline Expansion and Regulatory Progress
Nanoscope Therapeutics Inc., a clinical-stage biotechnology firm focused on gene therapies for retinal degenerative diseases and age-related macular degeneration (AMD), announced a successful End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) in September 2024. The meeting focused on the clinical program assessing MCO-010 for treating severe vision loss caused by Stargardt Macular Degeneration (SMD), paving the way for the progression of MCO-010 to a Phase 3 registrational trial.
Belite Bio applied to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) in March 2024 to initiate a clinical trial of Tinlarebant for treating adolescent Stargardt disease (STGD1). Named DRAGON II, this trial includes a Phase Ib open-label study and a Phase II/III global study. The Phase Ib study is tailored to evaluate the pharmacokinetics and pharmacodynamics of tinlarebant in Japanese adolescent STGD1 patients.
Comprehensive Pipeline Assessment
According to DelveInsight's assessment, the global Stargardt Disease pipeline constitutes 20+ key companies continuously working towards developing 20+ Stargardt Disease treatment therapies. Companies working in the treatment market include Ocugen, Belite Bio, Inc, Alkeus Pharmaceuticals, Biophytis, reVision Therapeutics, Inc, IVERIC bio, Inc., Nanoscope Therapeutics, Belite Bio, Kubota Vision, and others.
Emerging Stargardt Disease therapies in different phases of clinical trials include OCU410ST, Tinlarebant, ALK-001, BIO-201, REV-0100, Avacincaptad pegol, MCO-010, LBS-008, Emixustat, and others, which are expected to have a significant impact on the Stargardt Disease market in the coming years.
Disease Background and Unmet Need
Stargardt Disease is a rare genetic eye disorder that causes progressive vision loss due to the degeneration of the macula, the central part of the retina. It is the most common form of inherited juvenile macular degeneration and is primarily caused by mutations in the ABCA4 gene. Symptoms typically appear in childhood or adolescence and include blurred vision, difficulty seeing in low light, and central vision loss. There is currently no cure, but research into gene therapy and other treatments is ongoing.
The therapeutic pipeline spans various approaches including gene therapy, small molecules, and other innovative treatment modalities, with products categorized under different routes of administration including oral, parenteral, intravenous, subcutaneous, and topical delivery methods.
