BiotechTV - News SF Healthcare Week: Beam's CEO John Evans shares an update on based editing for alpha-1 antitrypsin deficiency, sickle cell disease, and more
Jan 12, 2026
John Evans, CEO of Beam Therapeutics, shares exciting advancements in base editing therapies for genetic diseases. He discusses potential accelerated approval for alpha-1 antitrypsin deficiency, highlighting regulatory feedback and a focus on confirmatory trials measuring lung and liver outcomes. Evans explains the advantages of base editing over other methods, especially for sickle cell disease, and teases future targets for liver conditions. With innovative approaches to LNP delivery, he emphasizes the platform's potential for customizable solutions in genetic therapies.
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Base Editing Corrects Alpha-1 Root Cause
- Beam's Beam 302 uses base editing to correct the single-letter DNA mutation causing alpha-1 antitrypsin deficiency.
- John Evans says biomarker changes they observed predict clinical benefit and support an accelerated approval path.
Engage Regulators Early For Accelerated Paths
- Pursue regulatory engagement early to enable accelerated approval when biomarker evidence is strong.
- Design an open-label cohort measuring biomarkers at 12 months to support accelerated approval, then run confirmatory outcome trials.
Biomarkers Then Confirmatory Outcomes
- Accelerated approval relies on surrogate biomarkers but requires later confirmatory trials measuring clinical outcomes.
- Beam plans to measure lung and liver function over time to confirm benefit after biomarker-driven approval.