Congratulations to our CEO, President and Founder, Dr. Emil Kakkis, on being honored as BIO’s 2019 Henri A. Termeer Biotechnology Visionary Award recipient for his transformative work in rare diseases. Read more.
Transforming good science
into great medicine
for rare genetic diseases.
Deep and diversified product pipeline
Developing multiple clinical-stage programs in parallel
Focused on serious rare and ultra-rare diseases
Working with patients and medical professionals to treat rare diseases
Dedicated to creating new treatments
Join a high-performance team focused on rare diseases
- For Patients