ACC 25: MyPEAK-1: Tn-201 Gene Replacement Therapy in Mybpc3-Associated Hypertrophic Cardiomyopathy скачать в хорошем качестве

ACC 25: MyPEAK-1: Tn-201 Gene Replacement Therapy in Mybpc3-Associated Hypertrophic Cardiomyopathy

First-in-human results from MyPEAK-1 show Tn-201 treatment in adults with Mybpc3-associated hypertropic cardiomyopathy was feasible and safe. Dr Milind Desai (Cleveland Clinic, Ohio, US) joins us onsite at ACC 2025 to discuss phase Ib/2a results from the first-in-human, non-randomized study investigating the safety and early efficacy of Tn-201, an adeno-associated virus serotype 9 gene replacement therapy, in adults with Mybpc3-associated hypertrophic cardiomyopathy (NCT05836259; Tenaya Therapeutics). The primary outcome measures of MyPEAK-1 were the number and severity of adverse events over the course of the study and the number of serious adverse events related to the study drug. Key findings in the first three patients showed the treatment was feasible, and safe. Interview Questions: 1. What is Mybpc3-associated HCM and what is the prevalence? 2. What are the current unmet needs in managing Mybpc3-associated hypertrophic cardiomyopathy, and why is this trial important? 3. Could you tell us about the mechanism of action behind Tn-201? 4. What was the study design and patient population? 5. What were the key findings, and were there any surprising results? 6. How should these findings impact clinical practice? 7. What further study is needed in this area? Recorded on-site at ACC in Chicago, 2025. Support: This is an independent interview produced by Radcliffe Cardiology. Visit Radcliffe Cardiology: https://www.radcliffecardiology.com/ This content is intended for healthcare professionals only. Radcliffe brings medical knowledge, insight and innovation to life for CV clinicians around the world, using our communications & creative expertise, our platforms and connections across the community to help transform theory into practice faster. Like us on Facebook:   / radcliffecardiology   Follow us on X: https://x.com/radcliffeCARDIO