Background – Genetic mutations and human diseases.Human evolution is encoded in our genes. Unfortunately, the process is not flawless. While “good” (gain-or-improvement of functions) changes in our genes take places in the evolutionary processes, “bad” (disease-causing) mutations also happen. These disease-causing mutations have been passed from generation to generation, in both genders and in all races.
A recent study using large, ethnically diverse clinical sample revealed that 24% of individuals were identified as carriers for at least one of 108 disorders, and 5.2% were carriers for multiple disorders. If we take more disorders into accounts, the carrier rate will be even higher. Therefore, genetic diseases concern every family and the society at large.
Genetic mutations not only happen in inherited diseases, they can also occur from damage to genes during a person’s life. These acquired mutations are the most common cause of cancer.
Gene therapy has changed dramatically in the 28 years since the first human gene transfer experiment in 1989. After decades of unfulfilled potential, this space is gaining tangible momentum. 2017 was the breakthrough year for gene therapy, as several gene and cell therapy treatments were approved in the U.S., offsetting the safety concern. From this year onwards, more gene therapy treatments will be commercialized for clinical use, helping particularly in addressing the significant unmet medical needs in a range of rare and challenging diseases.
Gene Therapy – How does it work?Gene therapy employs genes to treat or prevent diseases. It works by replacing a disease-causing faulty gene with a healthy copy of the gene, by editing and repairing the mutated gene, or by inactivating the mutated gene that is functioning improperly.
Advantages of gene therapy:
- Breakthrough treatments for challenging diseases, including inherited genetic diseases and other difficult-to-treat diseases, such as cancer.
- Gene therapy is a form of “Precision Medicine.”
- Aiming to treat diseases at the root level
- Long-lasting effect
Gene Therapy Industry News2017 was a landmark year for the gene therapy industry. For the first time in history, gene therapy was approved in the United States. In 2017, the U.S. FDA approved three gene therapy products in the fields of acute lymphoblastic leukemia, large B-cell lymphoma and inherited retinal disease caused by RPE65 mutations, respectively. Among them, cancer immunotherapy using CAR-T technology is a combination of gene therapy and cell therapy.
U.S. FDA approval brings first gene therapy to the United States
U.S. FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma
U.S. FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss
Our Research & DevelopmentReflectionBio is developing a gene therapy product for treating Bietti’s Crystalline Dystrophy (BCD), a devasting blinding disease. ReflectionBio has received Orphan Drug Designation from the U.S. FDA for our BCD gene therapy product. As the next step, we plan to advance BCD gene therapy into human clinical trial.
We expect to leverage our gene therapy platform technology to expand our R&D efforts into other disease areas.