Amicus Therapeutics

Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company focused on the development of advanced therapies to treat rare and orphan diseases. The company is headquartered in Cranbury, New Jersey, United States.

History

Amicus Therapeutics was founded in 2002. The company was established with the aim of developing innovative treatments for rare diseases, particularly those that are genetic in nature. Over the years, Amicus has grown through strategic partnerships and acquisitions, expanding its pipeline of therapeutic candidates.

Products and Pipeline

Amicus Therapeutics is known for its work in developing treatments for lysosomal storage disorders. The company's lead product is Galafold (migalastat), which is used to treat Fabry disease. Galafold is an oral precision medicine that targets the underlying genetic cause of Fabry disease in patients with amenable mutations.

In addition to Galafold, Amicus is developing a range of other therapies. These include:

• AT-GAA, a treatment for Pompe disease, which is currently in late-stage clinical trials.
• Gene therapy programs for various lysosomal storage disorders, including Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II).

Research and Development

Amicus Therapeutics places a strong emphasis on research and development. The company collaborates with academic institutions, research organizations, and other biopharmaceutical companies to advance its pipeline. Amicus is committed to leveraging cutting-edge science and technology to develop novel therapies for patients with rare diseases.

Partnerships and Collaborations

Amicus has formed several strategic partnerships to enhance its research and development capabilities. These collaborations have been instrumental in advancing the company's gene therapy programs and expanding its reach in the rare disease community.

Corporate Social Responsibility

Amicus Therapeutics is dedicated to corporate social responsibility, focusing on patient advocacy, community engagement, and ethical business practices. The company actively supports patient organizations and initiatives that aim to improve the lives of individuals affected by rare diseases.

Related Pages

• Biopharmaceutical company
• Rare disease
• Lysosomal storage disorder
• Gene therapy