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Home BIOTECH

Gene Therapy/Editing, Why is it so difficult?

disruptiveinvesting by disruptiveinvesting
July 15, 2021
Reading Time: 7 mins read
0
Gene Therapy/Editing

Twenty-one years ago this month, President Clinton declared “The Completion of the First Survey Of The Entire Human Genome” and the promise of future medical breakthroughs skyrocketed.  The narrative at the time was that genetic therapies were on the horizon and cures, especially for diseases where we had target geographies, would be manageable if not curable.  However, here we are over two decades later and with few exceptions, the only access patients can get to gene therapy is through clinical trials.  Why is this?  Why is this such a long journey?  Mapping of the human genome was a massive feat to be sure but in hindsight, we really were just scratching the surface and our understanding had just started.  We very quickly realized that we were just becoming adept at Checkers whereas nature was playing Three-Dimensional Chess.  Further, we had isolated and started to understand a static picture of a very dynamic system where a snapshot of where things were located was just not going to be enough.

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