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Science

At the forefront of breakthrough science

We accelerate the discovery and development of potential therapies in new and uncharted areas.

We adopt and adapt with agility.

We approach our science through the lens of patient need. We take a collaborative, multidisciplinary approach to discovery, applying adaptable thinking to foster innovation. By flexibly integrating new ideas and technologies, setbacks and uncertainties become opportunities for growth and creativity.

Infinite potential

We’re in. Learn how we strive to advance the next generation of potential medicines through innovative research. From San Diego to Cambridge, UK and everywhere in between.

We’re in. All in.

Our approach to science lets us see the bigger picture

Pipeline

We are advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery.

View our pipeline

Areas of innovation

We are investigating a wide range of technologies and modalities, including gene therapy, AI-driven protein engineering, protein manufacturing, RNA end-joining, and synthetic rescue.

Explore our areas of innovation

Our innovation engine

Our four laboratories are the driving force behind our research efforts and collaborate seamlessly across geographies. We are pursuing multiple solutions, technologies, and modalities in parallel in an effort to maximize impact and accelerate discovery. Data-driven decisions and synthesis of insights allow us to pivot quickly when needed and uncover new opportunities.

4 Global Research Centers

Our four laboratories are the driving force of our research efforts. We pursue multiple technologies and modalities in parallel, in an effort to maximize impact and accelerate discovery.

Independent thinking, unified global vision

One Research Council

Our Research Council evaluates science and works together to proactively plan our IND pipeline. Each year at our global symposiums, our teams come together to share knowledge and ideas, discuss ongoing projects, and lend support across sites and areas of expertise.

Charting the course ahead

Cross-site Collaboration

We foster a collaborative ecosystem where individual teams work closely within their own labs, then come together to share insights, cross-pollinate ideas, and refine approaches through new perspectives.

Our cohesive ecosystem

Business development

We actively seek opportunities to collaborate with forward-thinking teams across biotechnology and academia that share our sense of urgency and patient focus.

Partner with us

Clinical trials

To make a real-world impact, it’s vital that we understand real-life challenges. Our clinical trials are designed with input from patients to ensure their insights and needs are considered.

Explore clinical trials

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