Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities. […]
We enlist the best of the best - people with the right technical expertise and a relentless drive to solve real problems - and create an environment that empowers our teams to pursue bold, innovative science.
With this distinctive approach to drug discovery, we've produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an...