BioMarin Partners Swiss Biotech to Develop Cardiac Gene Therapies

US biotech BioMarin has partnered Swiss startup DiNAQOR to develop gene therapies to treat rare, inherited cardiac diseases.

DiNAQOR will initially receive an upfront payment from BioMarin, followed by developmental, regulatory, and commercial milestone payments and a percentage of royalties on later sales. No financial details of the collaboration were revealed by either company.

“We believe that by partnering with DiNAQOR for their lead clinical candidate we can accelerate our internal work that is already underway in genetic heart diseases,” a BioMarin representative told me. 

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“DiNAQOR emerged as the perfect match to turbocharge our program. We were just beginning, and they had already done a lot of work in the area.”

BioMarin currently has a broad pipeline of therapies for rare genetic diseases including six approved therapies and several in development such as a gene therapy for hemophilia A. BioMarin will work with the Swiss biotech on developing DiNAQOR’s lead candidate gene therapy, which it has licensed. This treatment targets a specific type of inherited hypertrophic cardiomyopathy, a condition that causes the cardiac muscle to enlarge and can lead to heart failure.

BioMarin will lead the development process, but “the DiNAQOR team will be an active, collaborative partner throughout the development process bringing in-house expertise in both cardiology and gene therapy,” according to Valeria Ricotti, Executive Vice-President & CMO at DiNAQOR, who previously worked at BioMarin as Medical Director of Clinical Sciences.

DiNAQOR was founded in 2019 by several biotech, cardiology and gene therapy experts, and has people based in the UK and US, as well as in Switzerland. Although it only launched last year, it already has two ongoing collaborations with University College London and Swiss biotech giant Lonza to develop cardiac-focused gene therapies.

There has been a revolution in gene therapy over the last few years, since the approval of eye gene therapy Luxturna. However, most such therapies focus on easier-to-target areas like the eye or target single gene disorders. Gene therapies focusing on cardiovascular disorders are less common.

Momentum for gene therapies, especially in cardiology, continues to build,” Ricotti told me. “We expect a rapid advancement of gene therapies into the clinic over the next several years with a focus on new vectors and potentially organ-specific delivery methods.”


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