Vertex to advance two AAT correctors to potentially treat rare, genetic disease
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Vertex Pharmaceuticals (NASDAQ:VRTX) will advance both VX-634 and VX-864 through the next stages of development as potential treatments for the rare disease alpha-1 antitrypsin deficiency (AATD).
The US FDA just cleared the company's IND to begin first-in-human testing of VX-634, a small molecule AAT corrector.

Vertex (VRTX) also said it will begin a 48-week phase 2 study of VX-864, another AAT corrector, to evaluate the impact of longer-term treatment on polymer clearance from the liver, as well as the resultant levels of functional AAT in the blood plasma.
AATD is a genetic disease characterized by a protein folding defect that can lead to liver and lung disease.
Check out why Seeking Alpha contributor William Meyers is bullish on Vertex (VRTX).