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MilestoneApr 1, 2026209 days ago

BEACON Phase 1/2 risto-cel sickle cell data published in NEJM

BEACON Phase 1/2 risto-cel sickle cell data published in NEJM, on Apr 1, 2026. A company milestone for Beam (risto-cel (ristoglogene autogetemcel, BEAM-101) for Sickle cell disease with severe VOCs): a step in the program rather than a decision or a data readout.

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DetailsCompany milestone
Company
Beam Therapeutics Inc. BEAM
Drug
risto-cel (ristoglogene autogetemcel, BEAM-101)
For
Sickle cell disease with severe VOCs
Stage
Phase 1/2 · an early study that checks safety and takes a first look at effect
When
Apr 1, 2026
Source note
Interim data from the Phase 1/2 BEACON trial of ristoglogene autogetemcel (risto-cel, formerly BEAM-101) in sickle cell disease with severe vaso-occlusive crises were published in The New England Journal of Medicine.
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Beam Therapeutics Inc.
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Trial resultsBEAMBeamH2 2026

Beam expects to report results from its Phase 1/2 study of Risto-cel (BEAM-101) for Sickle cell disease in the second half of 2026 (no exact date yet). Phase 1/2 is an early study that checks safety and takes a first look at effect.

More details
Entry
Updated BEACON Phase 1/2 data for risto-cel by year-end 2026 · Clinical trial results (readout / topline data)
Drug
Risto-cel (BEAM-101)
For
Sickle cell disease
Stage
Phase 1/2 · an early study that checks safety and takes a first look at effect
When
H2 2026 · the company gave a window, not a day
Source note
Company guidance: 'expects to report updated data for the BEACON trial by year-end 2026.' Dosing complete in all adult and adolescent patients.
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Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page

Trial resultsBEAMBeamH2 2026

Beam expects to report results from its Phase 1/2 study of BEAM-301 for Glycogen storage disease type Ia (R83C) in the second half of 2026 (no exact date yet). Phase 1/2 is an early study that checks safety and takes a first look at effect.

More details
Entry
BEAM-301 initial clinical data in glycogen storage disease type Ia · Clinical trial results (readout / topline data)
Drug
BEAM-301
For
Glycogen storage disease type Ia (R83C)
Stage
Phase 1/2 · an early study that checks safety and takes a first look at effect
When
H2 2026 · the company gave a window, not a day
Source note
Company guides initial clinical data 'in 2026'; only H2 2026 remains, hence the H2 tag.
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Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page

MilestoneBEAMBeamQ4 2026

Risto-cel (BEAM-101) BLA submission in sickle cell disease, as early as year-end 2026, expected in Q4 2026 (Oct–Dec) (no exact date yet). A company milestone for Beam (Risto-cel (BEAM-101) for Sickle cell disease): a step in the program rather than a decision or a data readout.

More details
Entry
Risto-cel (BEAM-101) BLA submission in sickle cell disease, as early as year-end 2026 · Company milestone
Drug
Risto-cel (BEAM-101)
For
Sickle cell disease
Stage
Phase 1/2 · an early study that checks safety and takes a first look at effect
When
Q4 2026 · the company gave a window, not a day
Source note
Company guidance: 'submit a biologics license application (BLA) for risto-cel as early as year-end 2026' (timing could slip into early 2027).
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Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page