DiNAQOR AG Developing and Bringing Gene Therapy to the Failing Heart

Dec 26, 2019 | Cardiomyopathy, Cardiovascular, Cell Therapy, Gene Therapy, Monogenic Cardiomyopathies, Viral Vector

DiNAQOR AG Developing and Bringing Gene Therapy to the Failing Heart

DiNAQOR AG is a global gene therapy platform building a ‘highway to the heart.’ In fact, for patients suffering from heart disease, the company is advancing novel therapies. Its lead preclinical program called DiNA-001 focuses on the treatment of MYBPC3-linked cardiomyopathy.

Based in Pfaffikon, Switzerland, with offices in London, UK, and Boston, MA, DiNAQOR has established a novel and innovate gene therapy platform supporting the evaluation of promising treatment for monogenic cardiomyopathies.

Who is DiNAQOR AG? Overview

Based in Switzerland, DiNAQOR is on a mission to develop gene therapy solutions that transform patients struggling with heart disease. They are developing advanced treatments for patients affected by genetic cardiomyopathies, a disease of the heart muscle that can lead to heart failure.

When were they founded?

April 2019, by a team of leading pharmaceutical and biotech executives and academics with deep cardiology and gene therapy expertise.

What Partnerships do they have?

They have strategic partnerships with Lonza (more on that to come) as well as the University Medical Center Hamburg-Eppendorf to advance their cardiac gene therapy platform pipeline. They have inked other partnerships, including Universite de NantesDeutshes Herzzentrum Berlin and Sirion Biotech.


The company is led by Johannes Holzmeister, MD, who earned his MD from Humboldt University, Berlin. He has led a number of different biotech groups and led international device group for heart failure patients at the University Hospital, Zurich. It should be noted they have put together an impressive scientific advisory board

What is their first therapeutic target?

Genetic cardiomyopathies are a group of disorders characterized by disease of the heart muscle—they are some of the most common genetic disorders and are a major cause of heart disease in all age groups, according to the Mayo Clinic Laboratories. Cardiomyopathy can be triggered by inherited genetic factors or by nongenetic (acquired) factors, such as infection or trauma. 

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.

Their initial focus is MYBPC3-linked cardiomyopathy—the human gene implicated in these diseases is cardiac myosin-binding protein C (MYBPC3), which is associated with thin filaments of sarcomeres.

What is their Core Intellectual Property?

Their DiNA-001 program and a local-regional delivery system for the heart. This will allow, upon validation via clinical trials, 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.

What is their Initial Preclinical Program?

Their preclinical program is called DiNA-001, an adeno-associated virus (AAV) gene therapy program for patients with MYBPC3-linked cardiomyopathy.


Their pipeline includes two targets at the “IND enabling” stage (meaning close to filing an investigatory new drug application), which, if approved, would allow them to commence Phase I clinical trials. This includes DiNA-001A (MYBC3—adult CM) and DiNA-001B (MYBC3—neonatal CM). In theory the partnership with Lonza should accelerate this effort.

What is the Nature of their Lonza Partnership?

Under the terms of the collaboration, Lonza will provide DiNAQOR with preclinical, clinical and commercial production support for the company’s lead preclinical program (DiNA-001). Lonza’s state-of-the-art cell- and-gene-therapy manufacturing facility in Houston, Texa,s will handle all product supply for DINA-001.

Lonza Background

Lonza is one of the world’s leading suppliers to the pharmaceutical, biotech and specialty ingredients markets. They focus on creating a healthy environment, promoting a healthier lifestyle, and preventing illness by supporting customers to deliver innovative medicines that help treat or even cure a wide range of diseases. Founded in 1897 in the Swiss Alps, Lonza is now a well-respected global company with more than 100 sites and offices and approximately 15,500 full-time employees worldwide. The company generated sales of CHF 5.5 billion in 2018 with a core EBITDA of CHF 1.5 billion.

Source: DiNAQOR AG


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