About AMT
Amsterdam Molecular Therapeutics is a world leader in the development of human gene based therapies. Our first treatment, Glybera® (alipogene tiparvovec), is a gene therapy for lipoprotein lipase deficiency (LPLD), an orphan genetic disorder. It is pending marketing approval in Europe. AMT also has a product pipeline of several gene therapies in development for hemophilia B, Duchenne muscular dystrophy, acute intermittent porphyria and Parkinson’s disease. AMT is located on the premises of the Academic Medical Center, University of Amsterdam, employs a staff of around 90 with extensive scientific and industry experience and is able to manufacture products on a commercial scale, meeting cGMP requirements.
Gene Therapy Opportunity
For many serious illnesses today, including inherited or genetic diseases, there is no lasting solution or cure available that addresses the cause of the problem. Existing therapies are limited to offering symptomatic relief at best.
Genetic diseases are caused by a gene mutation, leading to loss of function or diminished function. Many genetic diseases are rare; such diseases are called ‘orphan diseases’ and are debilitating and mostly lack effective therapy. Gene therapy inserts a functional copy of the gene into nuclei of cells of the patient so that the cells can regain their proper function. In addition to the treatment of hereditary diseases, gene therapy can be applied to other diseases, such as liver diseases, and disorders of the central and peripheral nervous systems and cardiovascular diseases, where the delivery of a therapeutic gene can render significant benefit to patients.
Lead Product
The marketing application for Glybera® is currently being reviewed in Europe. It has been developed to treat LPLD, a genetic orphan disease for which no drug therapy exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or total lack of functionality of LPL protein in patients. The LPL protein is needed to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess build up of chylomicrons can result in severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of the disease. In LPLD, patients often suffer recurrent attacks of pancreatitis.
Glybera® has orphan drug designation in the EU and the USA.
Strategy
AMT’s goal is to become a specialty biopharmaceutical company that develops gene therapies for diseases with a significant unmet medical need, and to market these independently or via partnerships.
Our programs are either targeted at orphan diseases, or are driven by their potential to replace existing products that provide sub-optimal solutions for substantial markets.
For products that are more suited for commercialization via partnerships, we aim to sign these relatively early, to generate revenues and provide non-dilutive funding. This can involve “disruptive” gene therapy programs aimed at replacing existing therapeutic products.
Platform
Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, AMT has been able to design and validate probably the world’s first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare diseases caused by one faulty gene, and allows AMT to pursue its strategy of focusing on this sector of the industry. AAV vectors are generally considered safe and have been tested in over 60 clinical trials worldwide to date.
