Vertex Pharmaceuticals, from Boston, MA, led in using smart drug design, a step away from mix-and-match chemistry, pioneering biotech.

Vertex Pharmaceutical

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Vertex Pharma, with a 1.75% stake, excels in cystic fibrosis treatment. Investors seek diversification for sustained growth beyond its billion-dollar CF success.

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Vertex faced setbacks with AATD treatments in 2020-21, causing a 35% stock drop, signaling challenges in diversifying beyond CF.

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Vertex and CRISPR Therapeutics made big strides treating blood disorders. Progress in their program is significant and noteworthy.

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Vertex and CRISPR Therapeutics got the green light for Casgevy, a sickle cell treatment. This win signals Vertex's success beyond cystic fibrosis.

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Vertex is set to launch Casgevy now, with nine approved treatment centers nationwide. Approximately 16,000 eligible patients could benefit from this gene-editing product.

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Vertex and CRISPR Therapeutics face slow revenue growth due to extended treatment periods. Goldman Sachs predicts Casgevy's peak revenue at $3.9 billion.

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Vertex pays CRISPR Therapeutics $200 million after FDA approval, keeping 60% of product profits per the profit-sharing deal.

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Vertex might introduce new products, like a pain management treatment. They're finishing a crucial trial, with results expected early next year.

Image Credits: Fierce Pharma