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?