Amicus Therapeutics

Patient-dedicated Biotechnology Company




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Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering high-quality medicines for people living with rare metabolic diseases.

With one rare disease medicine globally commercialized for Fabry disease, an investigational enzyme replacement therapy (AT-GAA) in late-stage development for the treatment of Pompe disease, and a robust gene therapy pipeline and growth platform for lysosomal storage disorders, Amicus Therapeutics is committed to changing the lives of people with these life-threatening conditions.

What we do today has its roots in 2005 when the employees of Amicus crafted a belief statement that would encompass our core values.

It reminds us of the reason we do what we do and outlines the fundamental beliefs that are crucial to understanding the passion and commitment behind Amicus.

We are proud to stand by this set of standards that showcase our bold nature and driven personalities.
We believe …

 … In the Fight to Remain at the Forefront of Therapies for Rare and Orphan Diseases
… In Our Future to Build Long-term Value for Our Stakeholders
… In Each Other to Foster Teamwork and Respect for Each Individual’s Contribution

Beyond our mission statement, we are guided by the programs we invest in, each of which must meet three key characteristics.

It has to be in the rare diseases for a devastating disorder, the technology has to have the potential to be first in class or best in class, and the clinical data must be able to provide meaningful benefit for patients.

As we pursue treatments for rare metabolic disorders, we maintain a personal and compassionate focus on patients, their caregivers, and families.

“This personal and direct relationship with patients embodies the culture of our organization and drives what we do very passionately every day,” says John F. Crowley, Chairman and CEO of Amicus. “Every biotech company says it’s focused on patients, but at Amicus we do it in a different way.

You see it when you go to our offices around the world where we have pictures of patients with the diseases we treat accompanied by narratives about their struggles, identities, and lives.”

Currently in Amicus’ development pipeline are therapies for Fabry, Pompe, and Batten disease.

In addition to these clinical programs, we have the ability to leverage our biologics capabilities and platform technologies to further expand our pipeline.

For example, the company is developing a gene therapy portfolio of intrathecal AAV programs for neurologic lysosomal disorders through a license agreement with The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital (NCH).

Amicus also has a broad gene therapy collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn).

Amicus’ dedication to patients goes hand-in-hand with a deep dedication to ethics and compliance. Acting with integrity and complying with law ensures that everything we do for patients meets the highest standards of safety and professionalism.

“As colleagues, we respect and trust each other,” says Patrik Florencio, Amicus’ Chief Compliance & Risk Officer.

“As friends, we go the extra mile to support each other in our mutual corporate objectives. And this cuts across all departments. It is that support, and understanding that compliance is a shared responsibility, that makes Amicus so special and its commitment to ethics & compliance so meaningful.”

Looking to the future, we want to engage in a journey of learning, adjustment, and focus. Fulfilling our vision requires extraordinary science, aligning priorities, and building a team of great people, and our passion unites us as we remain committed to helping those in need.

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