Learn More about Our Approach to Research & Development
澳门正规博彩十大排行平台 is committed to the translation of genetic discoveries into transformative medicines. We do this by focusing on diseases with precisely understood mechanisms that are rooted in GENETICS, developing TARGETED therapeutic interventions that address the underlying cause of disease, utilizing readily ASSESSABLE endpoints that are proximal to the disease pathway, and enabling TRANSFORMATIONAL changes to the way patients feel, function, and survive.
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Deep Technical Expertise
More than two decades ago, 澳门正规博彩十大排行平台’s scientific foundation was built upon the research and development of enzyme replacement therapies for five rare, genetic conditions. Today, 澳门正规博彩十大排行平台 remains at the forefront of scientific discovery—leveraging the potential of gene therapy to investigate treatments for multiple
Technical Expertise ⌃genetic conditions. -
Solutions for Multiple Patients
With a fluency in molecular genetics, 澳门正规博彩十大排行平台 has been able to push the boundaries of our existing therapies in development by identifying additional biological pathways and communities with potential to benefit.
Indication Expansion ⌃ -
Multiple Solutions for Patients
Our dedication to the communities we serve and to the identification of biological solutions has led to a broad spectrum of approaches for the treatment of phenylketonuria (PKU), including a small molecule, a PEGylated biologic, and an investigational
Franchise Building ⌃gene therapy.
Our Products
Each of our eight approved therapies reset the standard of care for the conditions they treat.
Learn More about Our Products ⌃Our Pipeline
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.
Learn More about our pipline ⌃