Shortly after jumping from academia to industry in 2015, David Altshuler helped answer an existential question for Vertex Pharmaceuticals. The Boston biotech was riding high off a series of blockbuster treatments for cystic fibrosis, and Altshuler joined as the chief scientific officer charged with placing its next big bets.
The result was a unique R&D strategy. It targeted a small set of diseases — including sickle cell and type 1 diabetes — and then used whatever tool or platform was needed to go after them, such as CRISPR-Cas9 for sickle cell, cell therapy for diabetes, or small molecules for pain. The CRISPR therapy will get an approval decision in December in what could be the first such treatment on the market.
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