BioMarin Pharmaceutical has signed a preclinical collaboration and license deal with Swiss gene therapy platform company DiNAQOR to develop novel gene therapies for the treatment of rare genetic cardiomyopathies.

DiNAQOR will get an undisclosed upfront payment and will stand to receive development, regulatory and commercial milestones on product sales apart from tiered royalties on global sales.

Initially, the license covers DiNA-001, the lead program of DiNAQOR. which is being developed for MYBPC3 hypertrophic cardiomyopathy (HCM). In addition to that, the firms will work together on various other pipeline programs of DiNAQOR.

Dr. Johannes Holzmeister – Co-Founder, Chairman and CEO at DiNAQOR said: “BioMarin is a global leader in rare disease research, development and commercialization, and their commitment to DiNA-001 is a powerful validation of DiNAQOR’s gene therapy platform.

“We believe our platform has many potential applications and this milestone agreement will enable us to invest in expanding our genetic medicine pipeline.”

BioMarin Pharmaceutical has been given the option to extend the license to include the additional programs on similar terms. In view of the long-term commitment to the partnership, BioMarin Pharmaceutical is investing simultaneously in the Swiss gene therapy platform company.

Jean-Jacques Bienaimé – Chairman and CEO of BioMarin Pharmaceutical said: “With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology.

“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.”

  BioMarin Pharmaceutical, DiNA-001, DiNAQOR, Dr. Johannes Holzmeister, gene therapy, Jean-Jacques Bienaimé, rare genetic cardiomyopathies