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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.
    com/news-releases/news-release -details/dicerna-announces-interim-results-phase-1-trial-belcesiran

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