Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose
- Friday, June 24, 2022, 7:04
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Serum TTR reductions were sustained at all doses tested with follow-up now reaching 12 months in the 0.1 and 0.3 mg/kg and six months in the 0.7 and 1.0 mg/kg cohorts Pharmacokinetic modeling and simulation indicated that an 80 mg fixed dose provides similar exposure to the 1.0 mg/kg…
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