M&A
Pharma's shopping list is looking specialized
Despite months of hopeful analyst forecasts, the world's major pharmaceutical firms have largely been loath to swoop in and acquire the many biotech companies that have been trading at depressed valuations for going on two years. And the deals that have materialized suggest pharma is prizing commercial potential over scientific promise.
The latest evidence comes from Bloomberg, which reported yesterday that large drugmakers are looking into bids for Apellis Pharmaceuticals, a roughly $8 billion company that recently won FDA approval for a pioneering eye treatment.
If Apellis gets acquired, it would fit in with Pfizer's $43 billion acquisition of Seagen, Sanofi's $2.9 billion takeover of Provention Bio, and Amgen's $27.8 billion buyout of Horizon Pharmaceuticals — cases of pharma companies spending cash on smaller firms with actual products, not cash-burning biotechs years away from the market.
Regulation
A milestone in genome editing
Partners Vertex Pharmaceuticals and CRISPR Therapeutics submitted their genome-editing treatment for FDA approval, pressing forward with what could be a curative medicine for sickle cell disease.
As STAT's Adam Feuerstein reports, the two companies outpaced their rivals at Bluebird Bio, whose gene therapy for sickle cell disease is expected to go before the FDA some time this year.
Vertex and CRISPR Therapeutics' treatment would be the first medicine based on CRISPR technology to reach the market. It works by editing a patient's own blood stem cells to produce high levels of fetal hemoglobin — the healthy, oxygen-carrying form of hemoglobin that is produced during fetal development but normally shuts down soon after birth. Higher levels of fetal hemoglobin in people with sickle cell disease are associated with reduced symptoms and improved outcomes.
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