Purposeful science – Lasting patient impact
We are building a deep and expansive pipeline of rationally designed gene therapy product candidates, pioneering a shift to treat not only rare but also prevalent diseases.
Our initial focus is on diseases of the heart and the nervous system, where we foresee a generational change in the treatment paradigm similar to what has happened in the treatment of cancers. This is because advances in science are identifying new genes associated with diseases of the heart and the nervous system, making a compelling case for genetic medicines. The heart and nervous system are also tissues where targeted delivery of the therapeutic genetic payload is currently inadequate. Our ART platform gives us an advantage to develop first-in-class or best-in-class product candidates.
Genetic medicines for the heart
We have applied ART to rationally design a new class of capsids with improved targeting of the heart muscle. In animal studies, our novel capsids given as a simple intravenous injection have been shown to reach almost all heart cells and in a uniform manner. In contrast, AAV9 reached less than 20% of cells at the same dose. The dose studied is typical of what is currently used in gene therapy trials for cardiac diseases in people. Reaching the predominant number of cardiomyocytes in a homogenous manner has been a long-standing limitation in the treatment of diseases of the heart such as genetic cardiomyopathies, life-threatening arrhythmias, and heart failure.
We are testing a number of product candidates in animal models to evaluate safety and efficacy in devastating diseases of the heart muscle and diseases affecting the heart rhythm.
Genetic medicines for the nervous system
We have applied ART to rationally design a new class of capsids that target the nervous system by crossing the blood-brain-barrier (BBB), a protective system that generally prevents capsids from reaching the brain. In animal studies, our novel capsids given as a simple intravenous injection have been shown to cross the BBB and reach the majority of neurons, which are the main cell types of interest in many diseases. In contrast, AAV9 reached less than 5% of neurons at the same dose. Crossing the BBB has been seen for decades as the holy grail in the treatment of diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and Huntington’s disease.
We are testing a number of product candidates in animal models to evaluate safety and efficacy in devastating diseases of the nervous system.
