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BioMarin Strikes Gene Therapy Partnership Targeting Rare Genetic Cardiomyopathies

Shanthi Rexaline

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is beefing up its gene therapy portfolio. 

The large-cap biopharma BioMarin said it has entered into a pre-clinical collaboration and licensing agreement with DiNAQOR, a gene therapy platform company headquartered in Switzerland, to develop gene therapies to treat rare genetic cardiomyopathies.

The licensing agreement will initially cover DiNAQOR's lead candidate DiNA-001, which is being evaluated for MYBPC3 hypertrophic cardiomyopathy, or HCM.

HCM is one of the most common genetic heart diseases, with about 500,000 patients diagnosed, and up to 60% of HCM cases have a genetic origin, BioMarin said.

It is estimated that 40% of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C, the company said. 

HCM affects the heart muscle, often increasing the risk of heart failure and life-threatening arrhythmias, and there is no currently available pharmacologic treatment option for the disease.

BioMarin, which itself is speculated to be an M&A target, said it has the option to extend the license to include DiNAQOR's other pipeline programs on similar terms.

BioMarin said it is also picking up a stake in DiNAQOR.

The financial terms of the deal were not disclosed.

BioMarin also reiterated its 2020 GAAP net income guidance of $20 million to $80 million, after factoring in this collaboration.

"With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology," BioMarin CEO Jean-Jacques Bienaimé said in a statement. 

BioMarin's BLA for AAV5 gene therapy candidate valoctocogene roxaparvovec has been accepted for review by the FDA with a priority review designation. The gene therapy, which is being evaluated for a bleeding disorder, has a PDUFA action date of Aug. 21.

BioMarin shares were down 1.44% at $86.85 at the time of publication Monday. 

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