Lonza and DiNAQOR AG, a global gene therapy platform company, have formed a collaboration to advance DiNAQOR’s preclinical programs for the treatment of cardiac myosin-binding protein-C (MYBPC3) cardiomyopathies, a genetic condition that can result in heart failure.
 
Lonza will provide DiMAQOR preclinical, clinical and commercial production support for the company’s lead preclinical program DiNA-001, an adeno-associated virus (AAV) gene therapy program for patients with MYBPC3-linked cardiomyopathy. Lonza’s cell-and-gene-therapy manufacturing facility in Houston, TX will handle all product supply for DiNA-001.
 
Through this partnership, DiNAQOR will be able to leverage Lonza’s extensive, dedicated teams and laboratories for viral-vector process-development, located in Houston.
 
In addition to its cardiac gene therapy platform, DiNAQOR is also developing a local-regional delivery system for the heart. This will allow the company to route gene therapy directly to the cardiac muscle maximizing biodistribution and transduction of the cardiomyocytes. This approach will look to minimize potential adverse effects of systemic gene therapy delivery.
 
“DiNAQOR has established an innovative gene therapy platform that will allow for the evaluation of this promising treatment for monogenic cardiomyopathies,” said Alberto Santagostino, senior vice president, head of cell and gene technologies, Lonza Pharma & Biotech. “DiNAQOR represents the truly trailblazing companies that we strive to empower in the cell and gene therapy space and we are fully committed to the DiNAQOR team as they seek to advance novel treatment options for people living with heart disease.”
 
Johannes Holzmeister, chairman and chief executive officer, DiNAQOR, said, “Lonza is a leader in the manufacturing of adeno-associated virus gene therapy vectors and is the optimal partner to help us rapidly advance and scale the production of DiNA-001 from early-stage clinical trials through commercialization. Precise and rapid genetic diagnostics, leading analytics, and an adequate product supply for all stages of clinical development and commercialization are necessary to bring these potentially transformational gene therapies to patients suffering from heart failure. We are excited to collaborate with Lonza on this critical initiative.”