Oscar Segurado, M.D., Ph.D.
Chief Medical Officer, ASC Therapeutics
Over the next five to 10 years, the management and treatment of hemophilia and other inherited blood disorders will experience a revolution. After decades of setbacks, gene therapy will become the treatment of choice for many patients. The replacement of defective genes will be achieved with a single intervention. In other words, these patients can be cured for life.
Gene therapy
ASC Therapeutics is a fast-growing biotechnology company focused on developing curative gene-based therapies for inherited blood disorders, initially focusing on hemophilia A and B and bet thalassemia. As a leading biopharma discovery company with over 11 years of experience in gene editing and stem cell technologies, we have created an end-to-end platform for gene therapy and gene editing. Our research and development efforts are focused on impacting patients’ lives with safe, life-changing, and affordable therapies.
Treatment for hemophilia A
Hemophilia A, also called factor VIII deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Patients are at risk for painful and/or potentially life-threatening bleeds from even modest injuries, and patients with the most severe form of the disease often experience painful, spontaneous bleeds into their muscles or joints.
Clinical studies have shown that gene therapy can achieve a permanent and successful amelioration of the bleeding episodes in adults with hemophilia, eliminating the need for prophylactic replacement therapy.
Solutions for children
However, these long-lasting therapeutic effects may not be reproducible in pediatric patients, those with the most to gain from effective bleed control, because of high turnover of the liver cells and loss of cells producing the missing coagulation factor. Our hemophilia A gene therapy will soon enter clinical trials after the company has offered best-in-class safety, low doses, fewer steroids, and sustained levels of clotting factor VIII in pre-clinical studies.
For the pediatric population, ASC Therapeutics is using a genome editing approach, a mechanism to rewrite the gene sequences. Gene editing has the potential to insert into a targeted position of the liver cell a gene that will produce the missing coagulation factor. Since this gene is fully integrated into the nucleus of the liver cell, even a rapid turnover of this cell will maintain the production of the missing factor after cell division.
Gene therapies are the most complex therapies ever developed, but will provide the most impressive outcomes ever imagined, a definitive cure for severe inherited diseases like hemophilia.