Intellia Announces Positive Clinical Proof-of-Concept Data for Redosing a CRISPR-Based Therapy with its Proprietary LNP-Based Delivery Platform
NTLAIntellia Therapeutics(NTLA) Newsfilter·2024-06-26 03:30

First-ever clinical data demonstrating redosing with an investigational in vivo CRISPR-based therapyFollow-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 Intelli ...