Gene Editing — Pipeline, Approvals, Sponsors

Targeted DNA modification: CRISPR-Cas9, base editing, prime editing. First approval (Casgevy for sickle cell) in 2023; expanding pipeline across hemoglobinopathies, primary hyperoxaluria, transthyretin amyloidosis.

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Marketed
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Filed
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Phase 3
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Phase 2
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Phase 1
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Total

Marketed leaders

No marketed leaders tracked.

Recent approvals (last 30 days)

No approvals in the last 30 days.

Upcoming PDUFA (next 180 days)

No upcoming PDUFA in next 180 days.

Top sponsors in this modality

No sponsor data.

Drug classes within Gene Editing

No class breakdown.

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Other modalities

BiologicsMonoclonal AntibodiesSmall MoleculesAntibody-Drug ConjugatesCell TherapiesGene TherapiesmRNA TherapeuticssiRNA TherapeuticsAntisense OligonucleotidesBispecific AntibodiesVaccines

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing