Intellia Therapeutics, Inc. presented new data demonstrating for the first time the potential for redosing with an investigational, in vivo CRISPR/Cas9 genome editing therapy. These data from the ongoing Phase 1 study of NTLA-2001, a single-dose treatment in development for transthyretin (ATTR) amyloidosis, were presented at the Peripheral Nerve Society Annual Meeting, taking place June 22-25 in Montreal, Canada. NTLA-2001 is currently being evaluated in patients with either ATTR amyloidosis with cardiomyopathy (ATTR-CM) or hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Development and commercialization of NTLA-2001 is led by Intellia as part of a multi-target collaboration with Regeneron.

Data from the Phase 1 trial has demonstrated that a one-time 55 mg dose of NTLA-2001 led to consistent, deep and durable reduction of serum TTR protein levels. This 55 mg dose was selected for further evaluation in the actively enrolling Phase 3 MAGNITUDE trial for ATTR-CM and the planned Phase 3 trial for ATTRv-PN.