Gene edting… editn… editing, time to fix our genome (1/3)

Over 6k genetic disorders are known. Only ~10% are “treatable”, and only four are curable. It is estimated that 1 in 50 people is affected by a known single-gene disorder.  Simple “text edits” in patients’ genomic information would be the holy grail for doctors and Biotechs. In this three-part series, we will unveil the world of gene editing and highlight the investment opportunities as it hits clinical trials.

Bottom line

The multi-Trillion-dollar genomic revolution may not be here yet, but we are getting very close to the multi-Billion-dollars foundation on which it will be based.

The last ten years have seen a flurry of sharp enough tools entering clinical trials: CRISPR, TALENs, zinc finger nucleases, base editing, prime editing, etc. As a result, cell therapy and in-vivo/in-situ gene editing are emerging as fields of interest for investors. 

Several roadblocks are still hindering the genetic medicine market from reaching its full potential but, at the same time, spurring technological innovation. Gene editing tools are newcomers, thus offering both hope and uncertainty. Discerning the most promising technologies is key to investors' performance.

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