BioMarin builds on gene therapy momentum with DiNAQOR deal in cardiomyopathy
BioMarin turns to external innovation to build its preclinical gene therapy pipeline
BioMarin’s gene therapy deal with DiNAQOR represents the first step in the company's plans to expand its gene therapy pipeline, while entering what’s becoming a hot cardiovascular indication: cardiomyopathy.
The companies partnered to develop gene therapies for rare cardiomyopathies in a deal that delivers DiNAQOR AG an upfront payment and an investment from BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) in addition to the potential for milestones and tiered royalties. In exchange, BioMarin received a license to DiNAQOR’s lead program and an option to extend