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Symbionyx Pharmaceuticals

Dallas, Texas, United States
Ownership
Private
Development stage
Other
01

Overview

Symbionyx Pharmaceuticals was a specialized biotechnology company focused on the development of pharmacological chaperones for the treatment of lysosomal storage disorders (LSDs). The company's core technology utilized small molecules to bind and stabilize misfolded enzymes, ensuring their proper trafficking to the lysosome and restoring enzymatic activity. In July 2005, Symbionyx was acquired by Amicus Therapeutics, a move that integrated its promising pipeline of chaperone therapies into Amicus's portfolio. This acquisition laid the groundwork for the development of Galafold (migalastat), the first oral precision medicine for Fabry disease. Symbionyx's research also contributed to programs targeting Gaucher disease and Pompe disease.

Therapeutic areas
Rare Diseases
Modalities
Small molecule
Industry
Biotech
02

Partnerships

Baylor Research Institute

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