The definitive guide to gene editing.
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Nasdaq: BEAM · Cambridge, Massachusetts

Beam Therapeutics

Founded by David Liu and colleagues to commercialise base editing — precision changes to single DNA letters without cutting.

Phase III Base editing platform

Pipeline

Where each disclosed programme stands. Stages come from the company's own disclosures on the date shown.

ProgrammeDiseaseTechnologyStage
BEAM-302 Alpha-1 antitrypsin deficiency Base editing DiscoveryPreclinicalPhase IPhase IIPhase IIIApproved
Risto-cel (BEAM-101) Sickle cell disease Base editing DiscoveryPreclinicalPhase IPhase IIPhase IIIApproved
BEAM-301 Glycogen storage disease type 1a Base editing DiscoveryPreclinicalPhase IPhase IIPhase IIIApproved
BEAM-103 Conditioning (enabling technology) Antibody DiscoveryPreclinicalPhase IPhase IIPhase IIIApproved

Pipeline as recorded in the Atlas on Aug 19, 2026. Companies revise pipelines frequently — verify against the company's own materials and regulatory filings.

In one paragraph

Beam was built around base editing, the technique its co-founder invented for changing one DNA letter into another without cutting the double helix. Its sickle cell therapy has finished dosing in trials with a licence application signalled for as early as the end of 2026, and its alpha-1 antitrypsin programme produced the first correction of a disease-causing mutation inside a living person.

Beam Therapeutics was founded in 2017 by David Liu, Feng Zhang and J. Keith Joung. Its portfolio spans ex vivo base editing (risto-cel for sickle cell disease) and in vivo base editing (BEAM-302 for alpha-1 antitrypsin deficiency, BEAM-301 for glycogen storage disease type 1a). BEAM-103, an anti-CD117 antibody, aims to replace genotoxic busulfan conditioning — potentially removing the largest source of toxicity in ex vivo cell therapy.

Sources

Not investment advice This profile is educational information compiled from public disclosures. It is not a recommendation to buy, sell or hold any security, and it is not a view on the company's prospects. Biotechnology is highly speculative and most clinical programmes fail. Full disclaimers →

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