Gene Therapy
Gene therapy is the use of genetic material as a treatment. This can mean the introduction, removal, or change of genetic material in the cells of a patient to treat an inherited or acquired disease. Successful gene therapy requires three main components: an optimized vector, a promoter, and a disease specific therapeutic transgene.
What is Gene Therapy?
Gene therapy transforms what was once mere wishful thinking into a groundbreaking reality: tackling inherited or acquired diseases at their root cause through the use of genetic material as a treatment. This innovative approach involves actions such as replacing or modifying a defective or missing gene, or introducing genetic material into specific cells of the patient. In this way, gene therapy marks a paradigm shift in healthcare, offering the potential to address areas of significant unmet medical need—where existing treatments have so far provided only limited or inadequate solutions for patients.
How does it work?
Gene therapies introduce functional genes into a patient’s cells, enabling the creation of essential proteins like enzymes or blood coagulation factors. This restores cell functionality and addresses the deficiency by targeting specific cells such as liver cells.
For safe, precise delivery, three key components are required: a vector, a promoter, and the transgene. Genes can’t be ingested or injected directly, as they would be cleared or degraded before reaching their target. A vector, typically a modified virus, acts as a carrier. Viruses are preferred because they naturally deliver genetic material efficiently. Before use, a virus’s disease-causing DNA is removed, leaving only its shell to carry healthy genes. Besides viral vectors, lipid nanoparticles are also being explored.
The transgene contains instructions to produce the therapeutic protein and is transported by the vector to the target cells. The promoter, a DNA segment before the transgene, acts as a switch controlling protein production in specific tissues or cells.
Once loaded, vectors deliver therapeutic genes into the patient’s cells, enabling proper function. This groundbreaking treatment can manage, halt, or reverse diseases with a single dose, providing lasting benefits.
What's 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. Furthermore, we collaborate with other leading gene therapy companies to expand the range of future application of our gene therapy platform.