BioMarin pens gene therapy pact with little-known Swiss biotech


BioMarin Pharmaceutical is boosting its early-stage pipeline by penning a deal with Swiss startup Dinaqor.

The deal, financials of which were not revealed, is a preclinical collab and license agreement that will see BioMarin tap into Dinaqor’s gene therapy platform, specifically to work on new therapies for rare genetic cardiomyopathies.

Dinaqor nabs an undisclosed upfront payment and is in line for biobucks, but the companies are keeping mum on figures. Given the early nature of the deal, one would expect the upfront to be low, but the biobucks could be reasonably substantial.

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The license initially covers Dinaqor's lead program, known as DiNA-001, which is targeting MYBPC3 hypertrophic cardiomyopathy (HCM). The pair will also work 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.” 

As part of this, BioMarin is also “simultaneously investing in DiNAQOR,” it said in a statement.

The biotech, which was only founded last year but already has a deal with University College London, focuses on gene therapies that can be safely delivered to the heart muscle, ensure transduction of the cardiac cells and limit the exposure of the therapy to other organs.

Its target, HCM, is diagnosed in about half a million patients around the world, and more than half (60%) of HCM cases have a genetic origin; 40% of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C.

HCM is a disease affects the heart muscle, causing the muscle to enlarge, meaning patients have an increased risk of developing heart failure and life-threatening arrhythmias.

"With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology," said Jean-Jacques Bienaimé, chairman and CEO at BioMarin. "This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies."

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This builds on BioMarin’s own gene therapy work in hemophilia, which could gain approval later this year.  

"We are thrilled to collaborate with the researchers at DiNAQOR to conduct this pioneering work on the development of gene therapies for inherited cardiomyophathies," added Lon Cardon, chief scientific 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."

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