And anytime the word “cure” appears in a sentence with a disease state, there’s cause for celebration. But we’ll have to wait a little while longer.
Philadelphia-based Sparks Therapeutics, which opened its doors in 2013, has acquired the developmental and commercial rights to a number of clinical and pre-clinical programs at Children’s Hospital of Philadelphia. The geneticists who have joined the Sparks team represent more than 30 years of research on gene therapy treatment, and the focus of one of those studies is hemophilia B.
This inherited bleeding disorder occurs when a person’s blood does not contain enough Factor IX, a clotting protein.
During one study, the patient received injections of viruses that had “corrected” versions of the deficient gene. What makes this so promising is the consistency of the results. One patient was able to discontinue self-infusions of factor—an action nearly unheard of just 10 years ago. But not all people with hemophilia B will respond well to this particular gene therapy, so the study will continue.