Key result
NTLA-2001 safely reduces serum TTR levels by ~90% at 28 days in ATTR-CM.
Why the study?
The study was designed to evaluate the safety, tolerability, efficacy, pharmacokinetics, pharmacodynamics, and serum TTR effects of IV NTLA-2001 for in vivo CRISPR/Cas9 editing in ATTR amyloidosis with cardiomyopathy.
Does IV NTLA-2001 safely reduce serum TTR levels in patients with ATTR amyloidosis and cardiomyopathy?
Population
12 patients with ATTR amyloidosis and cardiomyopathy
Comparison
IV NTLA-2001 at 0.7 mg/kg vs 1.0 mg/kg
Design
First-in-human in vivo trial
Follow-up
4-6 months
Authors
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Should not yet change ATTR-CM practice; hypothesis-generating for in vivo CRISPR TTR editing.
RCT (n=12)
Open-label
Randomized
Yes
Does IV NTLA-2001 safely reduce serum TTR levels in patients with ATTR amyloidosis and cardiomyopathy?
In a first-in-human trial, in vivo CRISPR/Cas9 editing with NTLA-2001 safely and substantially reduced serum TTR levels in patients with ATTR amyloidosis and cardiomyopathy.
Susy Kotit (2023) conducted an RCT in Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) (n=12). NTLA-2001 was evaluated on Changes from baseline serum TTR levels at 28 days. A single intravenous infusion of NTLA-2001 reduced serum TTR levels by a mean of >90% at 28 days in patients with transthyretin amyloidosis with cardiomyopathy, and was generally well tolerated.
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