Careers

Join a global team, moving at the speed of patient need.

Led by science, powered by people

Be part of a culture that thrives on being challenged.

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We’re proud to have earned:

#1 Best Place to Work, BioSpace 2026 (small biotech companies)
88% engagement score in 2025 employee survey

We’re proud to offer:

Market-leading pay + comprehensive healthcare
Flexible working model
Emphasis on growth and internal mobility
Opportunity to make a unique impact on patient lives

Driven by purpose

We’re fighting for people, not just against disease.

We take a different approach to Patient Advocacy, focusing on building enduring relationships and working hard to understand the needs of people living with genetic conditions. We encourage them to share their experiences via quarterly Patient Days, and our On Rare podcast.

A new type of biotech

High on standards, low on convention.

Our decentralized hub-and-spoke model lets us operate at scale with global infrastructure and commercial expertise, while empowering focused teams to act autonomously and follow the data to better outcomes.

How we work

Our culture is built on shared values. We move fast for the patients we serve, empowering independent thinkers to do the best work of their careers.

Our Values

Put patients first

We exist to help people living with genetic conditions. To do that, we listen to and work alongside communities to navigate challenges and co-create solutions, whether that means developing new treatments, shaping thoughtful trial designs, or providing other meaningful forms of support. Putting patients first means that compassion and understanding drive every step of our work. Always.

Think Independently

Experimentation is expected. We ask “Why?” or “Why not?”, challenging convention and approaching every problem with a rigorous, first-principles mindset.

Speak up

Great ideas come from a broad range of people (not just leaders) and should be worthy of consideration and testing. We encourage everyone to share their thoughts candidly so we get to the best possible answer.

Every Minute Counts

We work with urgency, giving autonomy to focused teams who can make informed decisions faster. Because the quicker we act, the sooner people are connected with meaningful treatments.

Let Science Speak

We’re driven by data and inspired by discovery. Our model is designed to give every potential medicine a fair chance to prove itself guided by clear criteria, grounded in science, and led by trust in data.