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VisitWill Intellia's CRISPR therapy enter Phase 2 trials for ATTR-CM by mid-2025?
Yes • 50%
No • 50%
ClinicalTrials.gov and Intellia press releases
Intellia's CRISPR Therapy Shows 90% Reduction in ATTR-CM Over 12 Months
Nov 18, 2024, 04:25 AM
Intellia Therapeutics has announced promising results from its ongoing Phase 1 study of its CRISPR therapy, nexiguran ziclumeran (nex-z), for treating transthyretin amyloidosis (ATTR-CM). The therapy demonstrated a 90% reduction in transthyretin levels over 12 months, indicating potential disease stabilization. This result is significant compared to Alnylam's siRNA therapy, which showed an 81% reduction. Additionally, Intellia's therapy showed gains in cardiomyopathy, further highlighting its efficacy.
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Yes • 50%
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Phase 2 Trial Completion • 25%
FDA Approval • 25%
Commercial Launch • 25%
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Successful reduction in serum TTR levels • 25%
High adverse events lead to study halt • 25%
Modifications required for safety • 25%
Study continuation without major issues • 25%
By Q1 2025 • 25%
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90% to 95% • 25%