Intellia Therapeutics Presents Data On Redosing With In Vivo CRISPR Therapy; 55 mg NTLA-2001 Dose Shows 90% Median Reduction In Serum TTR At Day 28; Well Tolerated Across All Patients; Potential Redosing Advantage For Future Therapies
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Biotechnology: Biological Products (No Diagnostic Substances)
Health Care
Biotechnology: In Vitro & In Vivo Diagnostic Substances
Health Care
- 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 study
- 55 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 Intellia's non-viral, lipid nanoparticle (LNP)-based delivery platform for future investigational therapies where a target additive effect is desired