Intellia, Regeneron post early data to highlight gene editing in ATTR amyloidosis cardiomyopathy
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Intellia Therapeutics (NASDAQ:NTLA) and Regeneron (REGN) announced Friday that NTLA-2001, a genome editing therapy targeted at heart-related issues in transthyretin (ATTR) amyloidosis, cut the serum levels of a key disease biomarker.
A rare disease caused by a genetic mutation, ATTR amyloidosis leads to the formation of a structurally abnormal transthyretin (TTR) protein, which builds up in different tissues. 
That leads to conditions such as cardiomyopathy (ATTRv-CM) and polyneuropathy (ATTRv-PN).
The announcement included data from 12 adult patients with ATTR amyloidosis with cardiomyopathy who received single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001.
As of Jul. 01, data cut-off, the interim data from the Phase 1 trial showed that NTLA-2001 caused serum transthyretin to decline, indicating mean reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses at day 28, respectively.
Patients tolerated both doses of the experimental therapy, and only two reported a transient infusion reaction, which was the only treatment-related adverse event.
âTogether with the previously reported data from the polyneuropathy arm of this landmark study, these results strongly suggest that NTLA-2001 could serve as a single-dose treatment regardless of disease manifestation,â NTLA Chief Executive John Leonard remarked.
In June, NTLA and REGN reported 12-month data for NTLA-2001 from a Phase 1 trial for patients with ATTRv-PN.