
for patients with
severe genetic diseases.

Setting the standard for gene therapy
Gene Addition Therapy: We’re building platforms with broad therapeutic potential because incremental improvements won’t do
For more than a decade, we’ve forged new paths for gene therapies and are improving and advancing at every step. We have the largest and deepest ex-vivo gene therapy data set in the world—driving the field forward. We are currently developing gene therapies for severe genetic diseases.