In vivo base editing therapy delivered by lipid nanoparticle to liver, correcting R83C mutation in glycogen storage disease type Ia, a rare genetic disorder affecting glucose metabolism.
Phase 1/2 dose-escalation trial ongoing in GSDIa; data readout expected late 2026.
Updated May 2025Checked 10 Oct0 updates this week
BEAM-301 converts disease-causing DNA directly. Unlike CRISPR, no double-strand breaks.
Lipid nanoparticles taken up by liver cells, where glucose defect causes disease.
Corrects mutation inside patient's liver cells, avoiding cell manufacturing.
| Spec | BEAM-301 |
|---|---|
| Target mutation | R83C (most common)R (reported) |
| Delivery format | Liver-targeted LNPR (reported) |
| Trial design | Phase 1/2 dose explorationR (reported) |
R reported by the company
Beam Therapeutics
Base editing, AATD, sickle cell
Base-editing company built on David Liu's lab work. Base editors change one DNA letter into another without cutting the DNA, fixing single-letter typos. Leads: BEAM-302 for alpha-1 antitrypsin deficiency and risto-cel for sickle cell.
