Beam Therapeutics Touts Encouraging Preclinical Data From Gene Editing Approach For Hepatitis B Infection

  • Beam Therapeutics Inc BEAM announced new preclinical data demonstrating the potential of its multiplex base editing approach to reduce hepatitis B surface antigen (HBsAg) expression and prevent viral rebound of hepatitis B virus (HBV) in vivo models. 
  • Base editors are designed to enable direct and irreversible conversion of a specific DNA base into another without inducing double-stranded breaks. 
  • In HBV-infected cells, cytosine base editors (CBEs) can target both integrated HBV DNA and the cccDNA minichromosome at multiple locations, introducing precise and permanent stop codons in the viral genome. 
  • The data builds on previously shared in vitro data, which demonstrated the ability of HBV-targeting gRNAs and mRNA-encoding CBEs to introduce stop codons in HBV DNA, leading to a substantial reduction of relevant HBV viral markers. 
  • Findings show that:
    • Base editing treatment led to a sustained >2 log10 IU/ml reduction of HBsAg observed in both LNP dose groups, compared to entecavir or control mice, in which no meaningful reductions were observed.
    • Base editing treatment led to a sustained 3 log10 copies/ml reduction in serum HBV DNA with no HBV viral rebound observed compared to the entecavir group in which serum HBV DNA was reduced following administration but rebounded after entecavir treatment was discontinued.
  • Price Action: BEAM shares are down 4.01% at $53.87 on the last check Monday.
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