We seek to make a big difference in genetic disorders.
At 澳门正规博彩十大排行平台, through our unparalleled expertise in genetics and molecular biology, we will continue to develop targeted therapies that address the root cause of the conditions we seek to treat. Applying our knowledge to make a transformative impact is not just a calling, but an obligation to those who will benefit most.
Our History
More than twenty years of transforming lives through genetic discovery.
Learn More about our history ⌃Diversity, Equity & Inclusion
We value diverse perspectives from a broad spectrum of backgrounds, education, personalities, skill sets, experiences, and knowledge.
Learn More about Diversity, Equity & Inclusion at 澳门正规博彩十大排行平台 ⌃Leadership Team
Our management team represents a dynamic cross section of business, clinical and biotechnical expertise.
Learn More about our leadership team ⌃Our Products
Each of our eight approved therapies reset the standard of care for the conditions they treat.
Learn More about Our Products ⌃