Novel Therapies for Stargardt Disease

Both gene-targeted and gene-agnostic treatments have been shown to restore ABCA4 function and impact broader retinal processes.

By: Cesar Estrada-Puente, MD, Paula C. Morales, MD, Ramiro S. Maldonado, MD

Retinal Physician October 1, 2025 Vol 22, Issue October 2025 Page(s): 19-22

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Stargardt disease is the most common inherited macular dystrophy, affecting 1 in 10,000 individuals due to ABCA4 gene mutations.
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Current therapies for Stargardt disease are under investigation, including ALK-001, tinlarebant, and emixustat, targeting toxic bisretinoid accumulation.
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The phase 2 TEASE-1 trial showed that ALK-001 significantly slowed atrophic lesion progression by 21.6% over 24 months compared to placebo.
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Tinlarebant demonstrated favorable outcomes in a phase 1b/2 trial, suggesting potential as a disease-modifying intervention in early Stargardt disease.
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Metformin is being evaluated for its ability to enhance lipid metabolism and reduce photoreceptor loss in Stargardt disease through a clinical trial.

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