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May 15, 2026 | Presentation

Discovery of RNA Exon-Editing Therapeutics Across Diverse Genetic Diseases via High-Throughput Screening, Enabling Novel Machine Learning Integration

Presented at the American Society of Gene & Cell Therapy 2026 Annual Meeting

By Kirk Burkhart, PhD

May 13, 2026 | Scientific Poster

MSH3 Pre-mRNA Splice Modulation in the Treatment of Repeat Expansion Disorders

Presented at the American Society of Gene & Cell Therapy 2026 Annual Meeting

By Akiko Doi, PhD

May 12, 2026 | Presentation

RNA Exon Editing for Stargardt Disease: Non-Clinical Development and First-in-Human Clinical Safety of ACDN-01

Presented at the American Society of Gene & Cell Therapy 2026 Annual Meeting

By Murray A. Abramson, MD, MPH

September 11, 2025 | Presentation

Treating Rett Syndrome via MeCP2 Editing

Presented at the Rett Genetic Medicines Summit 2025

By Kirk Burkhart, PhD

June 21, 2025 | Presentation

STELLAR Phase 1/2 Study: Rewriting ABCA4 RNA for the Treatment of Stargardt Disease

Presented at the Clinical Trials at the Summit 2025

Presented by Dr Christine Kay

June 10, 2025 | Presentation

Splicing the Dots: Potency Assurance Strategy for RNA-Editing Gene Therapies

Presented at the 2025 CASSS Cell & Gene Therapy Products (CGTP) Symposium

By John Kerwin, PhD

February 24-27, 2025 | Scientific Poster

MSH3 pre-mRNA splice modulation for the treatment of Huntington’s Disease

Presented at the 20th Annual Huntington’s Disease Therapeutics Conference

By Akiko Doi, PhD, et al.

September 10, 2024 | Publication

RNA Exon Editing: Splicing the way to treat human diseases

Published in Molecular Therapy: Nucleic Acids

By Akiko Doi, Conor Delaney, David Tanner, Kirk Burkhart, Robert D. Bell

May 8, 2024 | Scientific Poster

Exon Editing of ABCA4 RNA in Human Retinal Explants and Non-Human Primate Retina for the Treatment of Stargardt Disease

Presented at the American Society of Gene and Cell Therapy - 27th Annual Meeting

By Ting-Wen Cheng, PhD, et al.

May 20, 2023 | Presentation

Rewriting ABCA4 RNA for the Treatment of Stargardt Disease

Presented at the American Society of Gene and Cell Therapy - 26th Annual Meeting

By Robert D. Bell, PhD