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BioMarin and Skyline Therapeutics Announce Strategic Collaboration to Develop Novel Gene Therapies for Cardiovascular Diseases

Excerpt from the Press Release:

SAN RAFAEL, Calif. and SHANGHAI, Dec. 16, 2021 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Skyline Therapeutics (formerly Geneception), a gene and cell therapy company focused on developing novel treatments for unmet medical needs, today announced a multi-year global strategic collaboration for the discovery, development and commercialization of Adeno-Associated Virus (AAV) gene therapies to treat genetic cardiovascular diseases.

The partnership will leverage Skyline Therapeutics’ integrated AAV gene therapy platform based on its proprietary vector engineering and design technology and manufacturing capability to develop innovative gene therapies with a focus on genetic dilated cardiomyopathies (DCM), a group of progressively advancing, devastating diseases with no targeted treatment options.

Under the agreement, BioMarin and Skyline Therapeutics will collaborate on discovery and research through to an Investigational New Drug Application (IND). BioMarin brings experience in gene therapy development, cardiovascular biology and insights into genetic basis of diseases, and Skyline contributes its expertise in developing gene therapy products including vector engineering and design technology and manufacturing capabilities to this collaboration. Each company will advance the programs through clinical development in their pre-defined territories.  

In support of its R&D efforts for the collaborative projects, Skyline Therapeutics will receive an undisclosed payment associated with signing, comprising an upfront payment and an equity investment from BioMarin, and is eligible to receive pre-specified payments for R&D, regulatory and commercial milestones.

BioMarin will have the rights to commercialize therapeutic products resulting from the collaboration in its territories, including the United States, Europe, and Latin America, and Skyline Therapeutics will be responsible for commercialization in the Asia-Pacific region. In addition, Skyline Therapeutics will be eligible to receive royalty payments on future sales from BioMarin in its territories.

“We are thrilled to announce what we anticipate will be a fruitful collaboration at the interface between Skyline’s innovative approach to AAV vector engineering and design and our team’s proven expertise in creating and developing gene therapies,” said Kevin Eggan, Group Vice President, Head of Research and Early Development, from BioMarin.

“We are excited to partner with Skyline Therapeutics to tackle these genetic forms of dilated cardiomyopathy. This collaboration strengthens our leadership in cardiac gene therapy and extends our R&D collaboration to Asia, where a large number of patients suffer from these devastating diseases,” said Brinda Balakrishnan, Group Vice President, Corporate and Business Development at BioMarin. “We look forward to fostering this collaboration and bringing transformative medicines to patients worldwide.”

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