CRISPR–Cas9 hits its target in amyloidosis. Read the latest clinical trial analysis in The Clinical Pipeline, from CyrilPedia
have announced interim results
from the cardiomyopathy arm of their phase 1 trial for transthyretin amyloidosis. Patients treated with their CRISPR–Cas9-based gene-editing treatment, NTLA-2001, showed a dramatic reduction in levels of serum transthyretin . The announcement followed positive data published last year in from the hereditary polyneuropathy arm of the same trial. NTLA-2001 showed a good safety profile in both arms, but data on clinical efficacy are not yet available for either.
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