R&D leader Ver­tex ex­e­cutes a $420M cash gam­bit to dom­i­nate gene edit­ing field for Duchenne MD

Ver­tex sees a big fu­ture for it­self in us­ing gene edit­ing tech to rem­e­dy killer dis­eases like Duchenne MD, and it’s spend­ing $420 mil­lion in cash to­day to make it a near-term re­al­i­ty.

Af­ter the mar­ket closed Ver­tex an­nounced that it will buy gene edit­ing pi­o­neer Ex­on­ics — born in the lab of UT South­west­ern in­ves­ti­ga­tor Er­ic Ol­son — for $245 mil­lion while plunk­ing down an­oth­er $175 mil­lion to ex­pand its work with CRISPR Ther­a­peu­tics, the Swiss-based gene edit­ing out­fit that was in the first wave of star­tups in the field.

Last sum­mer, Ex­on­ics pro­vid­ed some in­trigu­ing de­tails to show how their gene-edit­ing ap­proach works in dogs. Dis­patch­ing a Cas9 scalpel with guide RNA in­side an AAV de­liv­ery ve­hi­cle straight to mus­cle cells, the re­searchers say that their ap­proach was able to re­store dy­s­trophin pro­duc­tion that reached up to 92% of nor­mal in a ca­nine mod­el for the dis­ease.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.