Beam’s Gene Editor Achieves First Ever Genetic Correction in AATD ##

Beam Therapeutics announced positive initial data from a Phase 1/2 trial of BEAM-302 for AATD1 8 19.
BEAM-302 is a liver-targeting lipid nanoparticle containing base editing components designed to correct the disease-causing PiZ mutation in AATD1 8.
This represents the first clinical evidence of precise correction of a disease-causing mutation by rewriting the genetic code in humans1 15.
A single dose of BEAM-302 led to durable, dose-dependent increases in functional alpha-1 antitrypsin (AAT) protein and decreases in mutant Z-AAT protein1 15.
At the highest dose tested (60 mg), BEAM-302 achieved mean total AAT of 12.4 μM at day 28, exceeding the protective threshold of 11 μM1 15.
BEAM-302 was well-tolerated with no serious adverse events reported across the first three dose cohorts1 15.
The company plans to continue dose escalation and expects to report additional data in the second half of 20251 8.
This approach has the potential to simultaneously treat both lung and liver manifestations of AATD by targeting the root genetic cause18 19.

This data provides clinical proof-of-concept for Beam's base editing technology as a potential one-time treatment for AATD, representing a major breakthrough in treating this genetic disorder1 18.