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

Dicerna Pharmaceuticals announced today that its investigational GalXC RNAi therapy belcesiran has achieved positive interim results in a phase 1 clinical trial

Belcesiran aims to target genes that produce abnormal AAT proteins to reduce AAT production in the liver

In this randomized, double-blind, placebo-controlled phase 1 clinical trial, healthy volunteers received a single injection of placebo or different doses of belcesiran

The results of the interim analysis showed that belcesiran reduced AAT protein levels by an average of 50% (1.

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

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Reference materials:

[1] Dicerna Announces Interim Results From Phase 1 Trial of Belcesiran for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease.