FDA Approves First Gene-Editing Cell Therapy For Sickle Cell Disease, An Inherited Blood Disorder
Portfolio Pulse from Vandana Singh
The FDA has approved two gene-editing cell therapies for sickle cell disease, Casgevy and Lyfgenia, for patients 12 years and older. Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses CRISPR/Cas9 technology and has also been conditionally approved in the U.K. for sickle cell disease and beta-thalassemia. Bluebird Bio's Lyfgenia uses a lentiviral vector for genetic modification and has received FDA approval ahead of its PDUFA date. However, shares of CRISPR Therapeutics, Vertex Pharmaceuticals, and Bluebird Bio fell after the announcement.
December 08, 2023 | 6:35 pm
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Bluebird Bio's Lyfgenia received FDA approval for sickle cell disease treatment, but the stock plummeted by 20.40% possibly due to the black box warning for hematologic malignancy.
The significant drop in Bluebird Bio's stock price could be a reaction to the black box warning included in Lyfgenia's label, which may raise concerns about the therapy's safety profile and affect its market adoption.
CONFIDENCE 90
IMPORTANCE 90
RELEVANCE 95
NEGATIVE IMPACT
CRISPR Therapeutics' Casgevy, co-developed with Vertex Pharmaceuticals, received FDA approval but shares dropped by 4.38%.
CRISPR Therapeutics' stock decline following the FDA approval might be attributed to profit-taking, market volatility, or concerns about commercialization and competition.
CONFIDENCE 85
IMPORTANCE 80
RELEVANCE 90
NEGATIVE IMPACT
Vertex Pharmaceuticals' Casgevy, a CRISPR/Cas9-based therapy for sickle cell disease, received FDA approval. The stock saw a slight decline of 0.72%.
Despite the positive news of FDA approval, the stock price of Vertex Pharmaceuticals decreased slightly, which could be due to market expectations already pricing in the approval or broader market conditions.
CONFIDENCE 85
IMPORTANCE 80
RELEVANCE 90