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

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 dose, where treatment with ntla-2001 resulted in 93% mean and 98% maximum serum ttr reduction by day 28 intellia to host investor event to discuss updated data from phase 1 study of ntla-2001, the first-ever systemically administered in vivo crispr investigational therapy today, friday, june 24, at 8:00 a.m. et cambridge, mass.
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