ACDN-01 / Ascidian Therap - LARVOL DELTA

RNA Exon Editing for Stargardt Disease: Non-clinical Development and First-in-Human Clinical Safety of ACDN-01 (ASGCT 2026) - No abstract available

Clinical • First-in-human • P1 data • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

Stargardt's Disease: Molecular Pathogenesis and Current Therapeutic Landscape. (PubMed, Int J Mol Sci) - "Small-molecule therapies target vitamin A dimerization (e.g., ALK-001), inhibit lipofuscin accumulation (e.g., soraprazan), or modulate the visual cycle (e.g., emixustat hydrochloride). Gene therapy trials explore ABCA4 supplementation including strategies like RNA exon editing (ACDN-01) and bioengineered ambient light-activated OPSIN...Trials like DRAGON (Phase 3, tinlarebant), STARLIGHT (phase 2, bioengineered OPSIN) show promise, but optimizing efficacy remains challenging. With the key problem of establishing genotype-phenotype correlations, the future of STGD1 therapy may rely on approaches targeting oxidative stress, lipid metabolism, inflammation, complement regulation, and genetic repair."

Journal • Review • Age-related Macular Degeneration • Gene Therapies • Inflammation • Inherited Retinal Dystrophy • Macular Degeneration • Metabolic Disorders • Ophthalmology • Retinal Disorders • ALK

Study to Evaluate ACDN-01 in ABCA4-related Retinopathy (STELLAR) (clinicaltrials.gov) - P1/2 | N=13 | Recruiting | Sponsor: Ascidian Therapeutics, Inc

New P1/2 trial • Age-related Macular Degeneration • Inherited Retinal Dystrophy • Macular Degeneration • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa