BEAM-301 / Beam Therap - LARVOL DELTA

Detection of G6PC RNA and protein activity in serum exosomes as pharmacodynamic biomarkers for GSDIa in vivo gene editing therapy (ESGCT 2024) - "BEAM-301 is a liver-targeting LNP formulation containing gRNA and mRNA components optimized to correct the R83C mutation...Additionally, the G6PC activity was detected and quantified in serum exosomes isolated from healthy donors, while no activity was observed in liver microsomes isolated from G6pc-R83C transgenic mice. Collectively, our approach highlights the potential utility of exosomal RNA and proteins as non-invasive pharmacodynamic biomarkers to assess response to treatment in in vivo base editing therapy for GSD1a."

PK/PD data • Preclinical • Metabolic Disorders • Severe Hypoglycemia

Beam Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights (GlobeNewswire) - "Beam plans to continue the dose-escalation portion of Part A of the ongoing BEAM-302 Phase 1/2 clinical trial and expects to report further data at a medical conference in the second half of 2025. The company plans to dose the first patient in Part B of the ongoing BEAM-302 Phase 1/2 clinical trial, which will include AATD patients with mild to moderate liver disease, in the second half of 2025. The company plans to continue dosing in the Phase 1/2 clinical trial of BEAM-301 in GSDIa."

P1/2 data • Trial status • Alpha-1 Antitrypsin Deficiency • Metabolic Disorders