Product: Enzyme Replacement Therapy for Gaucher’s Disease Developers: Dr. Yoseph Shaaltiel Company: Protalix Year of Foundation: 1993
Gaucher’s disease is not one of the world’s better-known diseases, however the Israeli company Protalix has devoted its efforts to developing a pioneering therapy for this debilitating disorder. Gaucher has become known as a “Jewish disease” due to the significantly higher rate of (mainly Ashkenazi) Jews with the disorder - as high as 1 in 450.
Gaucher’s Disease is a lysosomal storage disorder causing swollen organs (spleen, liver, abdomen), fatigue and easy bruising to severe neurological abnormalities and infant death.
In 1994, Dr. Yoseph Shaaltiel, a plant scientist who received his Ph.D. in Plant Biochemistry from the Weizmann Institute of Science, founded Protalix - a company that began in Israel’s northern town Kiryat Shmona, and set out to develop a biological plant-based protein for ERT (Enzyme Replacement Treatment), instead of the human or mammalian derived products that were
previously available.
The first manufacturing facility was constructed in May 2004 in Karmiel in the Western Galilee and was later expanded and upgraded at the end of 2009. Success was achieved in 2012 with the development of taliglucerae alfa – the first artificially produced plant cell-based human glucocerebrosidase - using a synthetic gene introduced into the carrot cell genome. The treatment was given the name ELELYSO. On May 1, 2012, the FDA approved ELELYSO for injection as ERT for the long-term treatment of adults with Type-1 Gaucher disease. Subsequent approvals were received from health authorities in Israel, Brazil and others.
The product’s distinction is that it is produced in a mammalian free environment which ensures safe and continuous production without the risk of the viral contaminations which exist in other production systems. Protalix changed the pharmaceutical world by introducing a safe, efficient and novel platform for the development of biological products and ELELYSO has changed the lives of thousands of patients with this rare disease, paving the way for additional plant derived products in the biomed industry.
The Protalix headquarters are still located in northern Israel in the town of Karmiel and the company currently employs about 230 workers.
