Question · Q4 2025
Luke Sergott asked for insight into the importance of numerical splice correction data for DM1, considering both clinical impact and regulatory perspective, and if a 20%+ correction remains a reasonable bar given the competitive landscape.
Answer
President and Chief Executive Officer Doug Ingram and Executive VP, Chief Scientific Officer, and Head of Research and Development Louise Rodino-Klapac stated it's too early to commit to a specific threshold. They will analyze the first cohort data, including muscle concentration and splice correction, and work with KOLs to inform Phase 3 design, noting prior FDA discussions are needed.
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