Biotechnology company developing therapies for rare metabolic diseases.
Amicus Therapeutics, Inc. is a pioneering biotechnology company dedicated to the discovery, development, and delivery of innovative medicines specifically targeting rare diseases. At the forefront of its offerings is Galafold, an oral precision medicine designed for adults diagnosed with Fabry disease possessing an amenable galactosidase alpha gene variant, validated through in vitro assay data. This commercial product exemplifies Amicus's commitment to advancing treatments that address the underlying genetic causes of debilitating conditions.
In its robust pipeline, Amicus Therapeutics is advancing AT-GAA, a groundbreaking therapeutic approach aimed at transforming the treatment landscape for Pompe disease. This novel treatment paradigm includes enzyme replacement therapies tailored to combat the complexities of Pompe disease, demonstrating the company's dedication to developing effective solutions for patients facing these challenging genetic disorders.
Further expanding its therapeutic innovations, Amicus Therapeutics is actively progressing CLN3, currently in Phase 1/2 clinical trials. This initiative evaluates the safety and efficacy of a single intrathecal administration of AT-GTX-502 gene therapy, designed in collaboration with leading institutions such as Nationwide Children's Hospital and the University of Pennsylvania, targeting patients with CLN3, a neurodegenerative disorder.
Founded in 2002 and headquartered in Philadelphia, Pennsylvania, Amicus Therapeutics continues to drive scientific advancements through strategic collaborations and license agreements, including partnerships with GlaxoSmithKline. The company remains steadfast in its mission to transform the lives of individuals affected by rare diseases by pioneering therapeutic innovations that address significant unmet medical needs.