Gene therapy: biopharma’s silver bullet?

October 1, 2019

Gene Therapy

The advent of gene therapy could disrupt the healthcare industry as we know it, redefining the traditional avenues that pharmaceuticals and biotechnology make profits.

Over the past five years, biopharma’s approach to disease treatment has changed dramatically. Now, in the post human genome era, scientific advances have enabled us to develop techniques to target the root cause of disease – the genes that encode for these malfunctioning proteins. Collectively, we refer to these therapies as Gene Therapy (GTx). This technology is far more precise and powerful than in the past and, in some cases, it can offer close to curative treatments in a single dose.

While there has only been a few GTx approved by the US Food and Drug Administration (FDA) to date, the research into such therapies has rapidly expanded, as exemplified by the Investigational New Drug (IND) applications that are filed to test therapies in clinical trials. So far, most of these therapies target orphan monogenic diseases (diseases caused by a defect in a single gene). However, we believe that GTx will ultimately be used to treat far more prevalent conditions like heart failure, diabetes and even Alzheimer’s. The rapidly expanding addressable market, cutting-edge scientific techniques, and remarkable healthcare benefits GTx can provide make it a theme that we believe is ripe with potential opportunities.

Amanda Birdsey-Benson, PhD, Director, Senior Research Analyst, Mellon

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