First In Vivo CRISPR Treatment Delivered Intravenously to a Patient
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Intellia Therapeutics, Inc announced that the first patient has been treated with NTLA-2001, which the company is developing as a single-course, potentially curative therapy for transthyretin amyloidosis (ATTR). Intellia’s global Phase 1 study is to evaluate NTLA-2001 for hereditary ATTR with polyneuropathy (hATTR-PN). Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader ATTR patient population of both polyneuropathy and cardiomyopathy patients.
“With today’s news, we’re entering a new era of potential genome editing cures for patients,” said Intellia’s President and Chief Executive Officer John Leonard, M.D. “Once we’ve assessed safety and established an optimal dose, we intend to rapidly initiate trials for the clinical manifestations of ATTR. NTLA-2001 may halt and reverse ATTR progression by producing a deeper, permanent TTR protein reduction for all patients – regardless of disease type – than the chronically administered treatments currently available.”
“Only a few short years ago, there were no treatments available for this devastating disease,” said Carlos Heras-Palou, M.D., founder and president of the United Kingdom ATTR Amyloidosis Patients Association (UKATPA), who also has hATTR. “Now, a cure for ATTR utilizing the groundbreaking CRISPR/Cas9 gene editing technology may be within reach.”
About the NTLA-2001 Clinical Program
Intellia’s global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). The trial’s primary objectives are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001. Patients receive a single dose of NTLA-2001 via intravenous administration. The study will enroll up to 38 participants (ages 18-80 years) and consist of a single-ascending dose phase in Part 1 and, following the identification of an optimal dose, an expansion cohort in Part 2. Visit clinicaltrials.gov (NCT04601051) for more details.
Enrollment is ongoing at a clinical trial site in the U.K. Intellia is submitting additional regulatory applications in other countries as part of its ongoing, global development strategy. The company is planning to rapidly move to pivotal studies enrolling both polyneuropathy and cardiomyopathy patients.
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first curative treatment for ATTR. NTLA-2001 is the first experimental CRISPR therapy to be administered systemically, or through a vein, to edit genes inside the human body. Intellia’s proprietary non-viral platform utilizes lipid nanoparticles designed to deliver to the liver a simple, two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 protein. Our robust preclinical data showing deep and long-lasting transthyretin (TTR) reduction following knockout of the target gene in vivo support NTLA-2001’s potential as a single course of treatment. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc.