Intellia Shares Updated Data On Gene Editing Durability In Rare Disease

Eight months after delivering promising biomarker data from one of the first CRISPR trials, Intellia Therapeutics Inc NTLA presented additional data from the Phase 1 trial for NTLA-2001 in transthyretin, or ATTR, amyloidosis.

• In the initial study, Intellia showed that a single infusion of its gene-editing therapy could lower the amount of toxic protein in six patients by an average of 52% or 87%, depending on the dose. 
• The new results showed that a slightly higher dose produced similar results. An even higher one could reduce protein expression by an average of 93% across six patients, with minimal side effects. 
• The most common adverse events across 15 patients in the study were mild symptoms such as rash and nausea. 
• One dose-limiting toxicity was observed in a patient who experienced vomiting, rated as a grade 3 adverse event.
• Intellia confirmed that the reduction in serum levels of the transthyretin (TTR) was maintained in patients after follow-up ranging from two months to 12 months post-treatment.
• Intellia leads the development and commercialization of NTLA-2001 as part of a collaboration with Regeneron Pharmaceuticals Inc REGN.
• Price Action: NTLA shares traded 8.32% lower at $90.63 during pre-market trading on Tuesday.