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.
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