BMN 293 / DiNAQOR, BioMarin, Lonza - LARVOL DELTA

BMN 293 – a novel gene therapy candidate for MYBPC3‐deficient Hypertrophic Cardiomyopathy (ESGCT 2023) - "BMN 293 intravenous administration to healthy primates at a high dose, without concomitant immunosuppression, was also well-tolerated and yielded uniform distribution of cMyBP-C throughout the heart. Together these results support further development of BMN 293 for MYBPC3-associated HCM."

Gene therapy • Cardiomyopathy • Cardiovascular • Gene Therapies • Heart Failure • Hypertrophic Cardiomyopathy

Impact of AAV-MYBPC3 Gene Transfer on Heart Structure and Function in Human and Mouse Models of Hypertrophic Cardiomyopathy (AHA 2023) - "A single IV infusion of BMN 293 to MYBPC3-/- mice resulted in early and sustained reduction in left ventricular hypertrophy and durable improvements in diastolic function."

Preclinical • Cardiomyopathy • Cardiovascular • Gene Therapies • Heart Failure • Hypertrophic Cardiomyopathy

BioMarin Announces Record Fourth Quarter and Full Year 2022 Total Revenues Driven by Strong Global Demand for VOXZOGO and Steady Growth of Enzyme Business (Biomarin Press Release) - "BMN 293 (formerly DiNA-001) for MYBPC3 hypertrophic cardiomyopathy (HCM)...BioMarin's goal is to file an IND for BMN 293 in the second half of 2023."

IND • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

BioMarin Announces Fourth Quarter and Full Year 2021 Financial Results and Corporate Updates (Biomarin Press Release) - "BioMarin's collaboration with DiNAQOR to create gene therapies including BioMarin's goal to file an IND for DiNA-001 in 2023."

IND • Cardiomyopathy • Cardiovascular

BioMarin Announces First Quarter 2021 Financial Results and Corporate Updates (PRNewswire) - "DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales."

Commercial • Preclinical • Hypertrophic Cardiomyopathy