Lonza and DiNAQOR AG Announce Strategic Collaboration to Advance Gene Therapy Programs for Patients with Monogenic Cardiomyopathies

November 26,2019
  • Strategic collaboration to focus on DiNAQOR’s adeno-associated virus gene therapy production system
  • Companies to partner on gene therapy process and analytical development for preclinical, clinical and commercial production of DiNA-001, DiNAQOR’s lead preclinical program
  • Process development and manufacturing to be located at Lonza, Houston (US), Center of Excellence for cell-and-gene-therapy manufacturing

Quote from Alberto Santagostino, SVP Head of Cell & Gene Technologies, Lonza Pharma & Biotech:

“DiNAQOR has established an innovative gene therapy platform that will allow for the evaluation of this promising treatment for monogenic cardiomyopathies. 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.”

Quote from Dr. Johannes Holzmeister, Chairman and Chief Executive Officer, DiNAQOR:

“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.”

Basel and Pfäffikon, Switzerland,  November 26, 2019  – Lonza and DiNAQOR AG, a global gene therapy platform company, today announced a strategic 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.

Under the terms of the collaboration, 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 state-of-the-art cell-and-gene-therapy manufacturing facility in Houston, TX (US) 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 (US).

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.

About Monogenic Cardiomyopathies

Cardiomyopathy is a disease of the heart muscle that can lead to heart failure. Approximately 50% of all cardiomyopathies are caused by a single-gene or monogenic defect. There are 1.7 million people in the European Union and the United States currently affected by a monogenic cardiomyopathy, 300,000 of these individuals have a defect in the MYBPC3 gene. There is currently no cure for patients living with genetic cardiomyopathies.

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