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    Home > Biochemistry News > Biotechnology News > Can reduce the level of pathogenic protein by 80%. The preliminary clinical results of RNAi therapy are positive

    Can reduce the level of pathogenic protein by 80%. The preliminary clinical results of RNAi therapy are positive

    • Last Update: 2021-07-28
    • Source: Internet
    • Author: User
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    Dicerna Pharmaceuticals announced today that its investigational GalXC RNAi therapy belcesiran has achieved positive interim results in a phase 1 clinical trial
    .
    Belcesiran is an RNAi therapy under development for the treatment of alpha-1 antitrypsin deficiency-related liver disease (AATLD)

    .
    AATLD is a rare genetic disease.
    Due to mutations in the SERPINA1 gene, α-1 antitrypsin (AAT) mutants misfold and accumulate in liver cells, which can lead to liver fibrosis, cirrhosis and hepatocellular carcinoma

    .

    Belcesiran aims to target genes that produce abnormal AAT proteins to reduce AAT production in the liver
    .
    The data of the interim analysis showed that after a single administration of belcesiran, serum α-1 antitrypsin (AAT) decreased in a dose-dependent manner

    .
    At the same time, belcesiran has acceptable safety profiles and is generally well tolerated

    .

    In this randomized, double-blind, placebo-controlled phase 1 clinical trial, healthy volunteers received a single injection of placebo or different doses of belcesiran
    .
    This interim analysis included injection doses of 0.
    1, 1.
    0, 3.
    0, or 6.
    0 mg/kg

    .

    The results of the interim analysis showed that belcesiran reduced AAT protein levels by an average of 50% (1.
    0 mg/kg), 69% (3.
    0 mg/kg) and 80% (6.
    0 mg/kg) compared with baseline

    .

    In terms of safety, no serious adverse events were found, and 3 moderate treatment-related adverse events (TEAE) occurred, but they were not related to belcesiran, and other TEAEs were mild
    .
    None of the participants treated with belcesiran in this analysis reported clinically significant changes in lung function or laboratory tests

    .

    Note: The original text has been deleted

    Reference materials:

    [1] Dicerna Announces Interim Results From Phase 1 Trial of Belcesiran for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease.
    Retrieved July 21, 2021, from https://investors.
    dicerna.
    com/news-releases/news-release -details/dicerna-announces-interim-results-phase-1-trial-belcesiran

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