DiNAQOR and BioMarin to develop gene theapies for rare heart diseases

DiNAQOR was founded in 2019 by several leading pharmaceutical and biotechnology executives academics with deep cardiology and gene therapy expertise. The company is focused on developing gene therapies for the heart, the company's lead preclinical program, DiNA-001 addresses Hypertrophic cardiomyopathy (HCM), one of the most common genetic heart diseases, with about 500,000 patients diagnosed with HCM worldwide.

The startup has signed a collaboration and licence agreement with BioMarin Pharmaceutical Inc., a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare genetic diseases. The license initially covers DiNAQOR’s lead program, DiNA-001 for MYBPC3 HCM.  Additionally, the companies will collaborate on several of DiNAQOR’s other pipeline programs, and BioMarin has the option to extend the license to include these additional programs on similar terms.  Reflecting the long-term commitment to the collaboration, BioMarin is simultaneously investing in DiNAQOR.

DiNAQOR will receive 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.  The company did not disclose financial terms.  BioMarin management reiterated its 2020 GAAP net income guidance of $20 to $80 million, inclusive of this collaboration.

“We are thrilled to collaborate with the researchers at DiNAQOR to conduct this pioneering work on the development of gene therapies for inherited cardiomyopathies,” said Lon Cardon, Chief Scientific Strategy Officer and Senior Vice President at BioMarin.  “We believe there is tremendous potential in combining our experience in gene therapy research and development with DiNAQOR’s in-depth knowledge of genetic heart diseases.”

(Press release/ran)