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

Posted: Published on November 29th, 2019

This post was added by Alex Diaz-Granados

BASEL,Switzerland and PFFFIKON,Switzerland, Nov. 26, 2019 /PRNewswire/ -- 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 DiNAQOR preclinical, clinical and commercial production support for the company'slead preclinical program DiNA-001, anadeno-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.

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."

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 CardiomyopathiesCardiomyopathy 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.

About LonzaLonza is one of the world's leading suppliers to the pharmaceutical, biotech and specialty ingredients markets. We focus on creating a healthy environment, promoting a healthier lifestyle and preventing illness, by supporting our customers to deliver innovative medicines that help treat or even cure a wide range of diseases.

Patients and consumers benefit from how we apply our manufacturing technologies to the healthcare, hygiene and fast-moving consumer goods environment and to developing preservation and protection materials.

Founded in 1897 in the Swiss Alps, Lonza today is a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide (at the end of 2018). The company generated sales of CHF 5.5 billion in 2018 with a CORE EBITDA of CHF 1.5 billion. Further information can be found at http://www.lonza.com.

About DiNAQORFounded in 2019, DiNAQOR AG is a global gene therapy platform company focused on advancing novel solutions for patients suffering from heart disease. The company's lead preclinical program, DiNA-001is focused on the treatment of MYBPC3-linkedcardiomyopathy. DiNAQOR is headquartered in Pfffikon, Switzerland, with additional presence in London, England and Boston, Massachusetts (US). For more information visit http://www.dinaqor.com.

Lonza Contact Details

Dirk OehlersInvestor Relations Officer Lonza Group Ltd Tel +41 61 316 8540 dirk.oehlers@lonza.com

Dr. Sanna FowlerHead of External Communications Lonza Pharma & Biotech Tel +41 61 316 8929 sanna.fowler@lonza.com

DiNAQOR Contact Details

Kellie WalshKWM Communications Tel 1-914-315-6072 kwalsh@kwmcommunications.com

Additional Information and DisclaimerLonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited ("SGX-ST"). Lonza Group Ltd is not subject to the SGX-ST's continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

SOURCE DiNAQOR

DiNAQOR: A global gene therapy platform company

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

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