Intellia Therapeutics (NTLA) Announces Positive Clinical Proof-of-Concept Data for Redosing a CRISPR-Based Therapy with its Proprietary LNP-Based Delivery Platform

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Go back to Intellia Therapeutics (NTLA) Announces Positive Clinical Proof-of-Concept Data for Redosing a CRISPR-Based Therapy with its Proprietary LNP-Based Delivery Platform

Intellia Announces Positive Clinical Proof-of-Concept Data for Redosing a CRISPR-Based Therapy with its Proprietary LNP-Based Delivery Platform

June 25, 2024 3:30 PM EDT

First-ever clinical data demonstrating redosing with an investigational in vivo CRISPR-based therapy
Follow-on dosing with a 55 mg dose of NTLA-2001 led to a 90% median reduction in serum TTR at day 28 in the three patients who previously received the lowest dose in the Phase 1 dose-escalation study55 mg follow-on dose was well tolerated across all patients While redosing is not planned for the NTLA-2001 program in transthyretin (ATTR) amyloidosis, a redosing option could be an important advantage of Intellias non-viral, lipid nanoparticle (LNP)-based delivery platform for future investigational therapies where a target... More