BEAM-302: Rewriting Genes, Restoring Health – A Breakthrough in AATD Therapy! 🔬🚀
Dr RR Baliga's "Got Knowledge Doc" Podkast
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Show Notes
🔬 BEAM-302 is a liver-targeting gene therapy using base editing to correct the PiZ mutation in Alpha-1 Antitrypsin Deficiency (AATD). 🧬 I
t increases functional AAT 🫁 while reducing toxic Z-AAT, showing up to 78% reduction at the 60 mg dose. 📊
The Phase 1/2 trial demonstrated dose-dependent efficacy, a well-tolerated safety profile, and no serious adverse events (SAEs). 🛡️
With ongoing dose escalation and expansion to liver disease patients, BEAM-302 could be a one-time curative therapy 🚀, with further data expected in late 2025. 📅