Beam Therapeutics (NASDAQ:BEAM) is trading ~3.2% higher in the pre-market on low volume after the company reported preclinical data to support the potential of its novel lipid nanoparticle (LNP) delivery approach for in vivo base editing in the liver and other tissues.
The company has used mRNA-encoding adenine base editor (ABE) and guide RNA to evaluate various LNP formulations and mRNA production processes to optimize LNPs to enhance in vivo liver editing and avoid immune stimulation in non-human primates (NHPs).
The findings include increased editing potency with 60% editing at 1.0 mg/kg. The formulations up to 1.5 mg/kg LNP were well tolerated with minimal to mild increases in transient liver enzyme elevations that were resolved by day 15 following the treatment.
Beam Therapeutics (BEAM) also revealed that it identified a group of LNPs for delivery of base editors to hematopoietic stem and progenitor cells (HSPCs). The administration at 1.0 mg/kg led to 40% expression of mRNA cargo in cells, according to a statement from the company.
The delivery approach is under evaluation for potential use in hemoglobinopathies and other genetic blood disorders.
The companies focused on gene editing rallied in late June after Intellia (NASDAQ:NTLA) posted positive early-stage data for its gene-editing candidate NTLA-2001 in transthyretin amyloidosis ("ATTR") showing the potential of in-vivo CRISPR genome editing in humans for the first time.