Gene Therapy
Gene Therapy involves delivering genetic material into a patient’s cells to address the causes of disease. There are different ways that gene therapy may potentially work to help people living with a disease, including: introducing an active copy of a gene into the body to help treat a disease, turning off a gene that is causing disease or replacing a faulty gene with a healthy copy of the gene.
What is Gene Therapy?
Gene therapy turns what has long only been wishful thinking into a reality: addressing an inherited or acquired disease at its cause by using genetic material as a treatment. This can mean, for instance, the removal or change of a faulty or missing gene, or the introduction of genetic material into certain cells of the patient. In this sense, gene therapy represents a paradigm-shift in how healthcare systems can address diseases as it has the potential to address areas of high unmet medical need, where current treatment options so far can only provide limited or insufficient care for patients.
How does it work?
Gene therapy is the use of genetic material as a treatment. Using the body’s own language, written in genes, gene therapy delivers specific instructions for the cells to start producing treatments in the place in the body where they are needed. This can mean the introduction, removal, or change of genetic material in the cells of a patient to treat an inherited or acquired disease. Gene therapy requires three main components: an optimized vector, a promoter, and a disease specific therapeutic transgene.
A vector, typically a modified virus, acts as a carrier.
Of the other two components, the transgene holds all the genetic information for the cells to deliver the therapeutic response. Once packed into the vector, it travels to the target cell and delivers the correct instructions. The promoter, a DNA segment before the transgene, acts as a switch controlling protein production in specific tissues. This treatment can manage, halt, or reverse diseases with a single dose, providing lasting benefits.
What is Bayer's Strategy in Gene Therapy?
In 2020, Bayer acquired the industry-leader and now wholly owned subsidiary AskBio Inc. (AskBio), a pioneer in gene therapy holding more than 750 patents relating to the technology field. AskBio specializes in the use of adeno-associated viruses (AAV). Our scientists are currently working on the development of multiple treatments that are intended to help patients suffering from some of the most debilitating diseases across multiple therapeutic areas. These include cardiovascular, metabolic and neurodegenerative diseases as selected examples.