Intellia Therapeutics says its Crispr-based treatment succeeds in pivotal trial [MSNBC.com]
Vertex Pharmaceuticals Incorporated (VRTX)
Last vertex pharmaceuticals incorporated earnings: 4/29 04:01 pm
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Source: MSNBC.com
Intellia TherapeuticsThe company's treatment usesNobel Prize-winning technologyIntellia said the one-time treatment reduced attacks by 87% compared with a placebo, meeting the study's main goal. Six months after treatment, 62% of patients were free from attacks and weren't using other therapies, Intellia said.The company described the safety and tolerability of the treatment as "favorable," reporting the most common side effects were infusion-related reactions, headaches and fatigue. Analysts were closely watching safety in the trial since a patient in a separate trial of a different treatment from Intellia died from liver toxicity."When you think about where we started with Crispr, just 12 years ago with some of the fundamental insights, I think there was a lot of talk about what might be possible, and we've had reports along the way in terms of milestones, but this is the first Phase 3 data in any indication with in vivo Crispr where you're actually changing a gene that causes disea
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