A major constraint in the development of effective drug treatments for chronic heart failure has been the lack of suitable cell-based assays and disease models with physiological relevance. Recently, Axiogenesis has developed and patented in Europe and Japan (EP1745144 and JP4814875) an in vitro disease model that will accelerate drug development for Hypertrophic Cardiomyopathy (HCM). The patent covers the use of this model for drug testing, toxicity testing and target discovery.

HCM is a disease with a high unmet need; currently 1 to 5 people in a 1000 are affected. Despite enormous efforts in health care, the associated mortality remains extraordinarily high as current therapies are far from effective.

The disease model developed by Axiogenesis entails inducing the diseased state of hypertrophy in human and animal stem cell-derived cardiomyocytes in vitro mimicking the diseased state in vivo. These diseased cells can then be compared to exactly the same cells without the disease to identify genes involved in HCM. Apart from elucidating the molecular mechanism of hypertrophy this allows the identification and validation of novel druggable targets. Axiogenesis has already performed first expression analysis identifying a host of potential novel targets.

The real power however is the possibility to use the same system to screen for drugs in vitro that inhibit the induction of HCM or reverse HCM. The assay is in a plate-based format with possibilities to increase throughput further. Axiogenesis' proprietary hypertrophy platform will fuel pharma pipelines for future cardiomyopathy therapy.

The usage of uniform, standardized and highly pure preparations of cells exhibiting a defined disease tate is the major advance within this model. Therefore, all effects can be very clearly attributed to one cell type exhibiting a defined cardiomyopathic phenotype.

This model is integrated in Axiogenesis' clinical modeling platform, that now also enables patient stratification for clinical phase-II studies.

Axiogenesis:

Axiogenesis' core business is in the area of iPS or ES stem cell-based assays and customized cell lines that enable drug development. These include cardiomyocytes, smooth muscle cells, endothelial cells and a wide range of customer-specific cell types (hepatocytes, olfactory neurons, melanocytes, etc.).

Axiogenesis
Felix von Haniel
+4922199881825
felix.haniel@axiogenesis.com