Jennifer Doudna and Fyodor Urnov have founded a company that will take on the challenge of editing rare diseases, starting with phenylketonuria.
The problem is well known: many diseases are theoretically treatable by correcting the corresponding genetic defects with the help of CRISPR tools, but doing so risks being difficult or even impossible because of regulatory rigidity and economic unsustainability. The case of the first newborn treated with a bespoke therapy developed in record time (KJ Muldoon) was a splendid proof of principle. But it left many parents of children with rare diseases asking: when will it be our turn? At the same time, specialists have long been asking: will investors return to viewing gene editing as a profitable approach worth investing in?
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Up and down, following the excitement for the latest scientific exploit or frustration for disappointing results. CRISPR is young but already knows how volatile is the market. “Preprint wipes millions off CRISPR companies’ stocks,” cries the March issue of 
The Swiss army knife is still the best analogy to describe what CRISPR can do, according to 
CRISPeR FRENZY 