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Intellia Therapeutics to Present the First-Ever Clinical Data From Patients Redosed with an Investigational In Vivo CRISPR Gene Editing Therapy at the Peripheral Nerve Society Annual Meeting 2024
- Data to offer insight into the safety and pharmacodynamics of redosing patients with a systemically delivered LNP-based CRISPR gene editing candidate
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"- Data to offer insight into the safety and pharmacodynamics of redosing patients with a systemically delivered LNP-based CRISPR gene editing candidate\nCAMBRIDGE, Mass., June 17, 2024 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today 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 (ATTR) 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 (LNP)-based delivery platform for future investigational therapies where a target additive effect is desired. Presentation Details Title: Activity of Follow-On Dosing for an Investigational In Vivo CRISPR-Based LNP Therapy in Transthyretin Amyloidosis Session: Richard A.C. Hughes Symposium – Clinical Highlights – Late Breaking Abstracts and Clinical Trials Date and Time: Tuesday, June 25, 2024, from 3:45 p.m. – 4:00 p.m. EST Presenter: Jorg Taubel, M.D., FFPM, FESC, Visiting Professor at King’s College London and Chief Executive Officer of Richmond Pharmacology About NTLA-2001Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. NTLA-2001 is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. NTLA-2001 is the first investigational CRISPR therapy to be administered systemically to edit genes inside the human body. Interim Phase 1 clinical data showed the administration of NTLA-2001 led to consistent, deep and long-lasting TTR reducti...