ACDN-01 / Ascidian Therap - LARVOL DELTA

Discovery of RNA exon-editing therapeutics across diverse genetic diseases via high-throughput screening, enabling novel machine learning integration (ASGCT 2026) - P1/2 | "While rational design has successfully advanced individual candidates such as ACDN-01 (currently in the STELLAR Phase 1/2 clinical trial for Stargardt disease, NCT06467344), the complexity of RNA secondary structure and splicing dynamics necessitates a more sophisticated approach to quickly achieve optimal potency and selectivity of novel RNA editing constructs...RNA exon editing offers a robust solution to these challenges, and our curated dataset enables integration of machine learning rules of RNA exon editor design, dramatically improving cycle times and predictive power. Our results demonstrate RNA exon editing is a scalable, highly specific alternative to traditional gene editing approaches, capable of surmounting the historical limitations of both standard AAV-mediated replacement and earlier generations of gene editors."

Machine learning • Genetic Disorders • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders

RNA Exon Editing for Stargardt Disease: Non-clinical Development and First-in-Human Clinical Safety of ACDN-01 (ASGCT 2026) - P1/2 | "By reducing mutant ABCA4 RNA while restoring functional protein expression, this approach addresses the fundamental cause of the disease. ACDN-01 holds the potential to halt progressive vision loss and improve visual function in patients with Stargardt disease and other ABCA4-related retinopathies."

Clinical • First-in-human • P1 data • Gene Therapies • Inherited Retinal Dystrophy • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

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