Intellia Therapeutics to present data from patients redosed with NTLA-2001

Intellia Therapeutics announced the acceptance of an abstract featuring redosing data from the Phase 1 study of NTLA-2001 has been selected for an oral presentation at the Peripheral Nerve Society Annual Meeting, taking place June 22 – 25 in Montreal, Canada. NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed to be a single-dose treatment for transthyretin amyloidosis. In the dose-escalation portion of the Phase 1 study, the initial three patients received the lowest dose of 0.1 mg/kg and subsequently received a follow-on dose of 55 mg. These data will be the first-ever clinical data from patients redosed with an in vivo CRISPR-based gene editing candidate and provide insight on the safety and pharmacodynamic effect. While repeat dosing is not planned for the NTLA-2001 program for ATTR amyloidosis, a redosing option could be an important advantage of Intellia’s non-viral, lipid nanoparticle-based delivery platform for future investigational therapies where a target additive effect is desired.