Asset profile

Exagamglogene autotemcel (Casgevy)

Gene therapy / editing

Approved / Phase 4Mechanism not established
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Approvedregulatory verdict

Evidence outlook

Approved: Sickle Cell Disease, Transfusion-Dependent Beta Thalassemia

Regulatory approval supersedes design-level evidence grading; the trials behind approval are preserved below as the drug's evidence history.

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Competitive landscape

17 competitors in this universe: 0 direct rivals (same mechanism and indication), 0 same-class, 17 same-indication.

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DrugStageModalityMechanismEvidence signalReported efficacygraded featured trials
Exagamglogene Autotemcel (Casgevy): this drugApprovedGene therapy / editing—
Crizanlizumab-Tmca (Adakveo)ApprovedMonoclonal antibodyP-selectin
Hydroxyurea (Droxia)Approved——
Hydroxyurea (Siklos)Approved——
Hydroxyurea (Xromi)Approved——
L-Glutamine (Endari)Approved—Amino Acid
Lovotibeglogene Autotemcel (Lyfgenia)ApprovedGene therapy / editing—
Mitapivat (Pyrukynd)Approved——
Voxelotor (Oxbryta)Approved——
SenicapocPhase 3——
Decitabine / Tetrahydrouridine (NDec)Phase 2—DNMT1 (DNA methyltransferase 1)
HBI-002Phase 2Small moleculeCarbon monoxide (CO)-based gas therapeutic; oral liquid CO formulation
EDIT-301Phase 1/2Cell therapy—
ITU512Phase 1/2——
Nula-CelPhase 1/2Cell therapy—
DISC-3405Phase 1——

…and 2 more. Research shows the complete landscape.

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Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.

Upcoming events

(3 total)

Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.

2027 (2)
June 2027 (2)
Phase 3Sickle Cell DiseaseActive not recruiting
Est. primary completion 2027-06-06 · NCT05329649 · CT.gov ↗
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Phase 3Sickle Cell DiseaseRecruiting
Est. primary completion 2027-06-09 · NCT05477563 · CT.gov ↗
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2039 (1)
September 2039 (1)
Phase 3Sickle Cell DiseaseEnrolling by invitation
Est. primary completion 2039-09-30 · NCT04208529 · CT.gov ↗
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See the full readout calendar ↗

All registered studies

Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.