Core Insights - Tenaya Therapeutics presented interim data from the MyClimb study, the largest noninterventional natural history study of pediatric patients with MYBPC3-associated hypertrophic cardiomyopathy (HCM), at the European Society of Cardiology Congress [1][2][5] Group 1: Study Overview - MyClimb study includes over 200 participants diagnosed with MYBPC3-associated HCM before age 18, with data collected from 27 centers across the U.S., Canada, Spain, and the UK [2][5] - The study aims to characterize the relationship between genotype and cardiac measures over time, initiated in 2021 [5] Group 2: Key Findings - 93% of participants had the nonobstructive form of HCM, which currently lacks approved treatment options [3] - Genetic inheritance patterns revealed distinct risk profiles among participants, with nearly all Homozygous group members dying or requiring heart transplants before age one [3] - Compound Heterozygous participants had a median diagnosis age of 2.9 years, with 63% experiencing heart failure-related hospitalizations [3] - Heterozygous children had a median diagnosis age of 6.5 years, with 27% facing heart failure-related hospitalizations [3] Group 3: Predictive Risk Factors - Left Ventricular Mass Index (LVMI) was identified as a significant predictor of risk, with every 10-unit increase associated with a 10% higher hazard of serious events [3] - Genetic profiles and LVMI may inform risk stratification and intervention strategies for pediatric patients [2][3] Group 4: Gene Therapy Development - TN-201 is a gene therapy designed to deliver a working MYBPC3 gene to heart muscle cells, with initial data from the first three patients presented at a prior meeting [7] - The therapy has received Fast Track, Orphan Drug, and Rare Pediatric Drug Designations from the FDA, indicating its potential significance in treating this condition [7] Group 5: Company Background - Tenaya Therapeutics focuses on developing curative therapies for heart disease, with a pipeline that includes TN-201 for MYBPC3-associated HCM and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy [8]