Beam Therapeutics' Formulated LNP Show Tolerance, Early Storage Stability In Animal Studies

  • Beam Therapeutics Inc BEAM has announced new preclinical data for novel lipid nanoparticle (LNP) formulations for in vivo liver editing. 
  • The Company says that continued optimization of LNPs has led to a substantial increase in liver editing potency, which is believed to be a clinically relevant dose for its lead LNP program, which will be finalized later this year. 
  • Beam's LNP delivery system for the liver increased editing potency in NHPs, demonstrating up to 60% editing at 1.0 mg/kg.
  • Minimal to mild increases in transient liver enzyme elevations were observed that were resolved by day 15 post-treatment.
  • Stable formulations with potency maintained at -20 and -80 ⁰C out to three months.
  • Additionally, Beam has identified a family of LNPs for delivery of base editors to hematopoietic stem and progenitor cells (HSPCs) in mice, with administration at 1.0 mg/kg leading to 40% expression of mRNA cargo in cells. 
  • Beam is evaluating the approach for potential application in hemoglobinopathies and other genetic blood disorders.
  • Read Next: WHO Issues Framework For Governance For Gene Editing Technologies.
  • Price Action: BEAM stock closed 0.54% higher at $95.47 on Wednesday.
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Posted In: BiotechNewsHealth CareGeneralBriefsGene EditingPreclinical Phase
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