Intellia Therapeutics’ and Regeneron Pharmaceuticals’ investigational in vivo CRISPR/Cas9 genome editing therapy showed promising results as a single-dose treatment for transthyretin amyloidosis in an ongoing phase 1 clinical trial.

The interim data on NTLA-2001 included 12 adult patients with transthyretin amyloidosis with cardiomyopathy. The treatment was administered through intravenous infusion. The data showed “profound” reductions in serum transthyretin, said the companies.

The phase 1 study is evaluating NTLA-2001 in patients with either transthyretin amyloidosis with cardiomyopathy or hereditary transthyretin amyloidosis with polyneuropathy.

A protocol amendment has been submitted to evaluate a fixed dose corresponding with enrollment across both arms expected to be completed by the end of 2022, subject to regulatory feedback, the companies said.