Amicus Therapeutics For Genetic Diseases

Amicus Therapeutics is highly passionate about its work. It is a global company in the field of biotechnology. This company is into advanced therapies for treating rare as well as orphan diseases. The company has a highly robust development pipeline that includes treatments for several genetic diseases in humans.

Migalastat is considered by Amicus Therapeutics as its lead product candidate (MarketWatch). This can be considered as personalized medicine. It is in the last stages of its development. This will be used for treating individuals who are suffering from Fabry disease. This would require genetic diagnosis. Another product candidate is SD-101. Even this is in its last stages of development. This will be the first therapy to be marketed. It can be used for the genetic connective tissue disorder known as Epidermolysis Bullosa (EB). Amicus Therapeutics is making use of its biologics along with the Chaperone-Advanced Replacement Therapy platform technologies. These are being used for developing several enzyme replacement therapy products. These can be used for Fabry disease, besides Pompe disease, as well as several other Lysosomal Storage Disorders.

The lead biologics program of Amicus Therapeutics is ATB200/AT2221. This is a Pompe disease ERT. It is to be administered along with a pharmacological chaperone.

Amicus Therapeutics offers three clinical programs. In addition, they can leverage their biologics capabilities along with platform technologies that can help in expanding their pipeline.

The biologics capabilities of Amicus Therapeutics helps to provide a unique tool that can be used for developing Enzyme Replacement Therapy products that can be used for LSDs. Presently, Amicus Therapeutics has novel ERTs already that can be used for Fabry as well as Pompe disease. Now the technology platforms can be used for creating future opportunities that can lead to novel ERT being developed for other LSDs.

Currently, Amicus Therapeutics is researching a potential protein replacement therapy. This will be used in preclinical studies for the CDKL5 deficiency. Any kind of genetic mutations on this CDKL5 gene can lead to CDKL5 protein deficiency. This disorder manifests itself as persistent seizures. These begin in infancy itself. It can lead to a severe impairment that can take place in neurological development.
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