CAMBRIDGE, Mass. - Intellia Therapeutics, Inc. (NASDAQ:NTLA), a pioneer in gene editing technologies, has announced significant findings from a Phase 1 clinical trial, showcasing the successful redosing of a CRISPR-based therapy. The study revealed a 90% median reduction in serum transthyretin (TTR) after administering a 55 mg follow-on dose of NTLA-2001 to patients previously given the lowest dose. This redosing was well tolerated among participants.
The trial, part of a broader effort to treat transthyretin (ATTR) amyloidosis, a rare and often fatal disease, demonstrated the potential of Intellia's non-viral, lipid nanoparticle-based delivery platform. While the NTLA-2001 program currently does not plan for redosing in treating ATTR amyloidosis, the option to redose could be beneficial for future therapies targeting diseases that require an additive effect.
ATTR amyloidosis leads to the accumulation of misfolded TTR protein, causing severe complications in various body tissues. Intellia's NTLA-2001 aims to be the first one-time treatment for the disease by inactivating the TTR gene. The Phase 1 trial's initial participants, who received a lower dose resulting in a 52% median reduction, were later offered a 55 mg dose after two years of observation. This subsequent dose led to a 95% median reduction from original baseline levels in serum TTR by day 28 post-treatment.
The interim data from the trial has been promising, with the 55 mg dose of NTLA-2001 showing consistent, deep, and durable TTR reduction. These results support the ongoing Phase 3 MAGNITUDE trial for ATTR-CM and the planned Phase 3 trial for ATTRv-PN. Moreover, the safety and pharmacodynamics observed after redosing align with those seen after the initial 55 mg dose, with patients experiencing favorable safety and tolerability.
Intellia, in collaboration with Regeneron (NASDAQ:REGN), leads the development and commercialization of NTLA-2001. The company's CRISPR-based platform is part of a multi-target discovery and development partnership aimed at delivering novel treatments for serious diseases.
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