Amicus Therapeutics is a worldwide biotechnology company dealing with innovative and advanced therapies to treat various deadly rare diseases. They have also developed treatments meant to combat human genetic disorders. The company’s current and lead product is the migalastat, a personalized medicine developed to treat patients with Fabry disease in accordance to their genetic diagnosis. Another late-stage developed product is the SD-101, a therapy for the Epidermolysis Bullosa (EB). EB is a sporadic but deadly genetic disorder of the connective tissue. Amicus Therapeutics is leveraging their Chaperone-Advanced Replacement Therapy (CHART) and biologics principle technologies to generate novel enzyme replacement therapy (ERT) products. The new products are for diseases such as Pompe disease, Fabry disease, and other Lysosomal Storage Disorders (LSDs).
The company’s lead biologics program so far is ATB200/AT2221, an ERT uniquely designed for Pompe disease which will be given in combination with a pharmacological chaperone. The ERT is developed by specifically targeting the mutated proteins, and this approach is devoted to gaining enhanced treatments for the rare genetic diseases. Amicus Therapeutics has the passion for science which motivates the company towards their success. They have different programs which include; Epidermolysis Bullosa program, Fabry Disease Program, Pompe Disease program and Preclinical Programs. At a recent Scientific Conference, the team presented their elementary clinical data concerning Pompe Disease. They also got to learn any latest research in the neuromuscular disease.
Amicus Therapeutics won an award in 2016 for their first drug acceptance and approval (YahooFinance). John Crowley, the chairman of the company, was the one who accepted the Innovator Award on behalf of the enterprise. Amicus also has an intern program that takes people from different parts of the world to learn and gain the experience across the various disciplines. The program helps many individuals and being global it is an efficient method to distribute their knowledge all over. Amicus Therapeutics also holds volunteer programs. They spent three days helping the Habitat for Humanity in Monmouth County; together with the communities, they developed better and affordable houses for people in the area. Amicus Therapeutics is a company which values the lives of individuals, and their fundamental goal is to make the world a better place.
More about Amicus Therapeutics at https://www.crunchbase.com/organization/amicus-therapeutics