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

Loading...
Loading...
  • 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.
Market News and Data brought to you by Benzinga APIs
Posted In: BiotechNewsHealth CareGeneralBriefsGene EditingPreclinical Phase
Benzinga simplifies the market for smarter investing

Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.

Join Now: Free!

Loading...