Amsterdam Molecular Therapeutics (AMT) B.V.

Meibergdreef 61
1105 BA Amsterdam

Phone: +31-(0)20-566 7394
Fax: +31-(0)20-566 9272
Stock symbol: AMT (NYSE


AMT aspires to develop innovative gene therapies in order to significantly improve the lives of people affected by serious, debilitating diseases.
Our research is mainly focused on developing cures for inherited diseases that affect small numbers of patients (orphan diseases). In addition, several of our programs allow us to target selected non-orphan diseases associated with serious morbidity and / or high mortality that can be treated with gene therapy.
AMT’s research efforts are intended to fill an unmet medical need for treatment in the following areas:

metabolic diseases
liver diseases
blood coagulation diseases
diseases of the nervous system
diseases of the eye
Gene therapy is a technique for correcting or replacing defective or missing genes that cause a disease. The advantage of gene therapy is its potential to cure a disease, rather than just treat the symptoms. With successful gene therapy, a long-lasting and possibly life-long effect can be accomplished in patients with just a single set of injections.