Serenity is unlocking the full potential of gene therapy.
Gene therapy is going to become the standard of care for inherited diseases, which includes hemophilia, inherited retinal diseases, and neurodegenerative diseases. In order to make that happen, the human immune system needs to be properly modulated to accommodate consistent efficacy. Approximately 50% of the population is already immune against the common virus vehicles that are used to deliver genes to target cells. All patients administered with virus vehicles will develop antibodies against the vehicles, preventing a chance for a critical second dose. At Serenity, we're unlocking the full potential for gene therapy through our novel immunomodulatory platform, CargoTek, by focusing on AAV gene therapy for hemophilia A.
Hemophilia A is a genetic disease that impairs the ability to form blood clots. Without the ability to form blood clots, bleeding episodes persist and can be life threatening. In order to prevent life-threatening bleeding episodes, patients with hemophilia A require IV administration of the coagulation factor VIII. Unfortunately, 30% of patients are unresponsive to this therapy. Gene therapy is an exciting new type of treatment that avoids continual administration in addition to offering an effective treatment option to a patient population that does not respond to IV-administered factor VIII.
Serenity Bioworks has large plans for the pharmaceutical industry. Serenity's CargoTek has a potential to enhance the long-term efficacy and safety profiles for gene therapy, resulting in safe and longer-lasting treatments for hemophilia A.