Research & Development

Our approach to R&D is rooted in a deep understanding of the underlying mechanisms of disease. We apply our expertise across multiple treatment modalities to address a broad range of serious medical conditions.

Where Insight, Passion and Innovation Converge

Our industry-leading R&D program has a track record of successfully ushering in new medicines built from the foundations of genetic insights. We bring together leading experts across disciplines, combining a wide range of subject matter expertise and technical skills, as well as diversity of experience. We then empower those teams to work collaboratively in pursuit of breakthrough science.

Pioneering Treatment Modalities

We got our start developing enzyme replacement therapies for ultra-rare genetic conditions. In the two decades since, we’ve significantly expanded our areas of expertise – while always remaining firmly grounded in leveraging genetic insights and mechanistic understanding to develop transformative medicines. Today, our treatment modalities include:

  • Biologics
  • Gene therapies
  • Oligonucleotides
  • Small molecules

Our Expertise Drives Our Areas of Focus

We focus on health conditions with known genetic bases and use these insights to precisely target the underlying mechanisms. Our scientific discoveries and expertise have led us to focus primarily on the following areas:

See how we’re applying our technical expertise across these areas of focus with our approved medicines and pipeline.

Science Stories