Clinical-Stage Biopharmaceutical Company

Princeton-born.
Purpose-driven.
Patient-first.

From our headquarters in Princeton, New Jersey, Amicus Therapeutics is developing and commercializing novel therapies for patients living with rare diseases — because every patient deserves a chance.

Explore Our Pipeline Our Science
Clinical Programs
7+
Therapeutic Areas
Rare Diseases
Headquarters
Princeton, NJ
Mission
Patients First
Our Science

Engineering therapies for diseases
that others have left behind

At Amicus, we combine deep expertise in protein engineering, chaperone medicine, and gene therapy to address the root causes of rare genetic diseases. Our platform approach allows us to pursue multiple therapeutic strategies across a portfolio of targets.

01

Chaperone Medicine

Small-molecule pharmacological chaperones that stabilize misfolded proteins, restoring their function at the cellular level for lysosomal storage disorders and beyond.

02

Enzyme Replacement

Recombinant enzyme therapies designed to replace missing or deficient enzymes, addressing the underlying biochemical defect in metabolic diseases.

03

Gene Therapy

AAV-based gene replacement strategies aimed at providing durable, potentially curative treatment for inherited disorders with unmet medical needs.

04

RNA Targeting

Novel approaches to modulate gene expression at the RNA level, expanding our toolkit for diseases where traditional protein-based strategies fall short.

Pipeline

A portfolio built on
scientific rigor

Our pipeline spans multiple therapeutic areas and modalities, reflecting our commitment to addressing rare diseases through diverse scientific approaches.

Approved

Myozyme® / Lumizyme®

Pompe Disease

Enzyme replacement therapy for patients with glycogen storage disease type II.

Phase 3

AMG-301

Mucopolysaccharidosis

Pharmacological chaperone therapy targeting underlying protein misfolding.

Phase 2

Gene Therapy Programs

Rare Genetic Disorders

AAV-mediated gene replacement strategies for inherited metabolic diseases.

Early Stage

Novel Targets

Undisclosed Indications

Preclinical programs leveraging our platform technologies for new therapeutic areas.

Laboratory research facility
Our patients are not statistics. They are individuals, families, and communities who deserve every scientific advantage we can bring to bear.
— Amicus Therapeutics
Our Impact

Serving patients where
the need is greatest

Rare diseases affect millions of people worldwide, yet many receive little attention from the pharmaceutical industry. Amicus was founded on the conviction that the size of a patient population should never determine the value of a therapy.

Rare
Disease Focus
Global
Patient Reach
Novel
Therapeutic Approaches
Learn About Our Mission
About Amicus

A Princeton company with
a global mission

Founded in 2002, Amicus Therapeutics is headquartered in Princeton, New Jersey — a community renowned for its scientific excellence and innovation ecosystem. Our team brings together world-class scientists, clinicians, and industry veterans united by a single purpose: improving the lives of patients with rare diseases.

We operate with the understanding that rare diseases demand rare dedication. Every program in our pipeline is guided by deep scientific insight, patient-centric development, and an unwavering commitment to delivering therapies where they are needed most.

Amicus Therapeutics Princeton headquarters exterior

Scientific Excellence

Rigorous, platform-driven research that pushes the boundaries of what's possible in rare disease therapy.

Patient-Centered

Every decision we make is informed by the needs and experiences of the patients and families we serve.

Global Perspective

Rare diseases know no borders. Our approach is globally informed, with development and access strategies that reach patients worldwide.

Integrity & Transparency

We hold ourselves to the highest standards of ethical conduct, scientific honesty, and operational transparency.

Get in Touch

Let's connect

Whether you're a patient seeking information, a healthcare professional, a potential collaborator, or a member of the press — we'd love to hear from you.

Address
1 Cedarbrook Ter
Princeton, NJ 08540-7407

Send us a message

Is this your business?This is my business