Gene Therapy:


We have built a focused pipeline of innovative gene therapies and have established clinical proof-of-concept in our lead indication, hemophilia B.


Millions of patients rely on recurrent medical care to help manage their life-long chronic ailments, at significant cost and, depending on the disease, with little chance of sustained success. Gene therapy has the potential to be a one-time administration of a gene that produces a therapeutic protein long-term. This approach may provide treatment solutions for diseases for which there is currently no cure or therapy, or improve treatment for chronic diseases where current drugs involve involve burdensome administration or frequent re-administration, or limited efficacy.