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NTLA : Study Of NTLA-2001 On Transthyretin Amyloidosis Shows Serum TTR Reductions Remained Durable

Intellia Therapeutics Inc. (NTLA) and Regeneron Pharmaceuticals Inc. (REGN) announced additional positive interim data from an ongoing Phase 1 study of their lead investigational in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis.

The updated interim data from the study demonstrated that deep serum TTR reductions remained durable after a single dose.

According to updated data, the 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.

"Based on the interim data shared today, we believe NTLA-2001's potential to be a transformational treatment for patients with ATTR amyloidosis is becoming clearer," said Intellia President and Chief Executive Officer John Leonard.

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