We have built a focused pipeline of innovative gene therapies and have established clinical proof-of-concept in our lead indication, hemophilia B.
A large number of serious diseases result from a defect in a single gene, but gene therapy is not limited to monogenic diseases alone. 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. 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.
We believe that most research and development efforts in gene therapy have focused on monogenic diseases, which include hereditary diseases. Although these diseases are severe, their cause is generally understood and less complex than diseases that involve more than one dysfunctional or missing gene. There are opportunities to apply gene therapy in diseases caused by more complex pathology in which one particular protein plays a crucial role in the causation of the disease. In such indications, such as some liver diseases, disorders of the central nervous system and cardiovascular diseases, it may be possible to halt or eradicate the disease with a gene therapy that promotes the natural production or function of the relevant protein.