It’s always exciting when two giants of their fields join together to accomplish something. When Pixar and Disney joined forces to bring us the best animated films since… ever, the world rejoiced. When Batman and Superman teamed up to bring about a DC universe to compete against Marvel’s universe, moviegoers salivated with anticipation (but then the announcement that Ben Affleck would be Batman tempered our excitement). When Disney acquired the rights to the Star Wars franchise, fans of the original movies felt a new hope that a good Star Wars movie could or even would be made. There seems to be a reoccurring theme with Disney being one of the giants of an industry.
Regardless, two major pharmaceutical companies are teaming up to develop a gene therapy for people suffering from hemophilia A.
Sangamo Therapeutics and Pfizer, Inc. have joined forces to combat this rare blood disorder using a gene therapy rather than the more traditional factor replacements that have been the hallmark of hemophilia treatments for years. The human trials are only just beginning, so approval is still a little ways off. That being said, the U.S. Food and Drug Administration already granted the treatment Fast Track and Orphan Drug statuses, which should streamline the approval process somewhat.
People with hemophilia have abnormal or excessive bleeding episodes because their bodies do not contain enough of the coagulating agents, called factors, or the factors are not functioning properly. Various treatments have been used ever since the causes of hemophilia were identified in the 1960s.
This new gene therapy, known as SB-525, introduces a synthetic form of the clotting agent into the bloodstream that will promote the development of more factor. If successful, the trial could lead to more trials and eventual approval by the FDA. This particular version of the therapy is for hemophilia A, which affects approximately 16,000 people, nearly all men and boys, in the U.S. However, similar treatments may become available for others with hemophilia B and other bleeding disorders.
