Landscape #2: Future of Gene Therapy

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Introduction

Gene therapy is a very powerful therapeutic modality that is gaining momentum with researchers, investors, big pharma, regulators and even payors. And like cell therapy, gene therapy will allow researchers to harness the power of cells to enable more complex and dynamic treatments to serious diseases. However, the potential of current therapies on the market or in the clinic is limited to rare monogenic diseases. For the most part, these therapies introduce a wildtype gene to counteract loss of function mutations. This has the potential to cure debilitating diseases, and will be a huge success for modern medicine, however small patient sizes and intense competition may limit commercial success. Still, these first-generation gene therapies are only a drop in the bucket compared to the potential future applications of gene therapy.