BioMarin and Skyline Collaborate to Develop Novel Gene Therapies for Cardiovascular Diseases

BioMarin Pharmaceutical and China-based Skyline Therapeutics entered 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.

“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 of corporate and business development at BioMarin. “We look forward to fostering this collaboration and bringing transformative medicines to patients worldwide.”

Dilated cardiomyopathy (DCM) is a common cause of heart failure and end-stage DCM, which often leads to heart transplantation. Despite improvements in pharmacotherapy and care, the five-year survival rate of DCM is only about 50 percent. Hundreds of thousands of patients suffer from the genetic forms of DCM in U.S., EU, China, and Japan. More than 50 genes associated with DCM have been identified, accounting for 40-50 percent of familial DCM cases. Many of these genes encode proteins with important known functions in cardiomyocytes related to cytoskeletal, sarcomere, and nuclear envelope biology. Our aim is to correct the pathways altered by these genetic contributors to DCM through AAV based gene therapy, in each case addressing the root cause of the disease.

“Dilated cardiomyopathy is a serious cardiac disorder in which structural or functional abnormalities of the heart muscle can lead to complications such as arrhythmia and heart failure, resulting in substantial morbidity and mortality. Mutations in many genes are associated with the development of DCM, among other etiologies for the disease,” said Jay Hou, chief scientific officer at Skyline Therapeutics. “Together with BioMarin’s team we have identified a number of critical genes associated with DCM. We are delighted to work closely with BioMarin and apply our AAV vector technology to interrogate these new targets and develop novel treatments for DCM patients.”

Brinda Balakrishnan, group vice president of corporate and business development at BioMarin

Author: Rare Daily Staff