Vertex Pharmaceuticals (NASDAQ: VRTX) has built a billion-dollar cystic fibrosis (CF) treatment empire, but investors have been looking for something more. They've hoped to see the company broaden its work into other areas to add an extra lift to long-term growth.

When Vertex announced the failure of two candidates to treat alpha-1 antitrypsin deficiency (AATD), an inherited disorder affecting the lungs and liver, back in 2020 and 2021, investors took the news particularly hard -- seeing this as a sign Vertex was struggling to expand beyond CF. The stock sank about 35% from the first AATD failure to a low point in 2021.

Months later came a spark of hope. Vertex, along with partner CRISPR Therapeutics, announced significant progress in its program to treat blood disorders. Today, the dreams of these companies, patients, and investors have come to fruition. Vertex and CRISPR Therapeutics just won a regulatory nod for exa-cel, to be sold as Casgevy, for the treatment of sickle cell disease. With this approval, Vertex proved something big to investors: It can, indeed, succeed beyond CF. Does this make the biotech a buy? Let's find out.

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Source Fool.com