Gene Therapy in PKP2 ACM Clinical Trials

Gene Therapy in PKP2 ACM Clinical Trials Part of the Living with SADS Webinar Series Sponsored in part by Lexeo Therapeutics, Tenaya Therapeutics, and Rocket Pharmaceuticals ???? Before watching this webinar, we recommend viewing our two previous sessions with Dr. Andrew Landstrom on gene therapy basics: What it is and how it works! ???? Key Takeaways from "Gene Therapy in PKP2 ACM Clinical Trials" with Dr. Andreas Barth • PKP2 mutations are a common cause of Arrhythmogenic Cardiomyopathy (ACM), particularly involving the right ventricle. • Gene therapy aims to correct or compensate for the underlying PKP2 defect at a molecular level. • Clinical trials are now underway to assess the safety and effectiveness of these gene-based therapies. • Patient eligibility, informed consent, and long-term monitoring are key components of trial participation. • Early-phase trials are focused on safety—future phases will assess clinical benefit. • Participation in trials contributes to advancing potential treatments for future generations. • Collaboration between researchers, families, and advocacy groups like SADS is vital to the success of rare disease research. ????️ Featured Speaker: Dr. Andreas Barth Medical expert in cardiovascular gene therapy and clinical trial development, presenting an overview of emerging therapeutic approaches for patients with PKP2-related ACM.