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BioMarin, DiNAQOR To Develop Gene Therapies For Rare Genetic Cardiomyopathies

BioMarin Pharmaceutical Inc. (BMRN) said Sunday that it has reached a preclinical collaboration and license agreement with DiNAQOR AG to develop novel gene therapies to treat rare genetic cardiomyopathies. The company did not disclose financial terms.

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.

BioMarin reiterated its 2020 GAAP net income guidance of $20 million to $80 million, inclusive of the collaboration.

The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy. Hypertrophic cardiomyopathy is one of the most common genetic heart diseases.

In addition, 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.

BioMarin is simultaneously investing in DiNAQOR, reflecting the long-term commitment to the collaboration.

Hypertrophic cardiomyopathy affects the heart muscle, causing the muscle to enlarge.
Hypertrophic cardiomyopathy patients have an increased risk of developing heart failure and life-threatening arrhythmias.

There are no approved pharmacological treatment options available that address the underlying disease biology of Hypertrophic cardiomyopathy and invasive surgery or heart transplantation may be the only options available for patients with advanced disease.

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