RARE Daily

BioMarin and Dinaqor Collaborate to Develop Gene Therapies for Rare Genetic Cardiomyopathies

May 4, 2020

BioMarin Pharmaceutical has entered into a preclinical collaboration and license agreement with gene therapy platform biotech Dinaqor to develop novel gene therapies to treat rare genetic cardiomyopathies, chronic diseases of the heart muscle.

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. Financial terms were not disclosed.

“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,” said Lon Cardon, chief scientific strategy officer and senior vice president at BioMarin.

The license initially covers Dinaqor’s lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (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.  Along with the collaboration, BioMarin is making an equity investment in Dinaqor.

Hypertrophic cardiomyopathy (HCM) is one of the most common genetic heart diseases, with about 500,000 patients diagnosed with HCM worldwide. Up to 60 percent of HCM cases have a genetic origin, and it is estimated that 40 percent of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C.

HCM causes the heart to enlarge. HCM 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 HCM and invasive surgery or heart transplantation may be the only options available for patients with advanced disease.

Dinaqor is focused on developing gene therapies for the heart that can safely deliver gene therapies to the heart muscle, ensure transduction of the cardiac cells, and limit the exposure of the therapy to other organs.

“The deal with BioMarin is a powerful validation of its gene therapy platform, says Johannes Holzmeister, co-founder, chairman, and CEO at Dinaqor. “We believe our platform has many potential applications and this milestone agreement will enable us to invest in expanding our genetic medicine pipeline.”

Photo: Lon Cardon, chief scientific strategy officer and senior vice president at BioMarin.

Author: Rare Daily Staff

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