Gene therapy is going to become the standard of care for inherited monogenic diseases. This includes hemophilia, inherited retinal diseases, and neurodegenerative diseases. Despite early promising results, the human immune system needs to be properly modulated to accommodate long-term efficacy. Approximately 50% of the population is already immune against the common virus vehicles that are used to deliver genes to target cells. Further, nearly all patients will develop antibodies against the vehicles after their first dose, 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 severe cases require daily IV injections of coagulation factor VIII. Gene therapy is an exciting new type of treatment that promises a substantial improvement in the standard of care. Instead of daily-administered IV injections, patients would get effective treatment once every year.
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.