On a mission to redefine gene therapy

A research strategy focused on impact

We know the potential gene therapy has—that when every component works together in just the right way, the results can be transformative. Halting disease progression. Reducing symptoms. Changing lives.

That’s why we’re optimizing every component of our gene therapy candidates while expanding our focus to more prevalent diseases with high unmet needs. Toward more impact, and brighter futures.

Our pipeline

Building on our success with a groundbreaking gene therapy candidate for Gaucher disease, we’re moving from rare diseases toward more widespread conditions.

Disease area

Research

IND-enabling

Phase 1/2

Phase 3

Worldwide rights

Toward a new standard of care

Toward a disease-modifying treatment

Toward a first-in-class gene therapy

Toward more prevalent conditions

Worldwide rights:

Toward a new standard of care

Worldwide rights:

Toward a disease-modifying treatment

Worldwide rights:

Toward a first-in-class gene therapy

Worldwide rights:

Toward more prevalent conditions

What’s next: We’re developing more programs with potential to expand into larger disease areas, including certain forms of dementia and heart failure.

Our programs

Partner with us

We know that partnership and collaboration can help us spur gene therapy forward. We work with researchers and clinicians around the world, our peers in the healthcare industry, and patients and advocacy groups to advance our work and bring more impact to more people. Interested in collaborating?

Get in touch