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Asset / Drug

Casgevy

Drug and asset intelligence for Casgevy with 3 indexed intelligence signals across regulatory, clinical, competitive, and market developments.

Signals
1
Catalysts
2
Stage
NDA/BLA

Sponsor company

Vertex Pharmaceuticals →

Mechanism of action

Gene Therapy

Recent signals(1)

RegulatoryJuly 2, 2026

FDA Approves Casgevy for Young Children with Sickle Cell Disease

The FDA has granted supplemental approval for Casgevy for young children with sickle cell disease and transfusion-dependent β thalassemia.

Upcoming milestones(1)

PDUFA Date

Vertex Pharmaceuticals — Casgevy

First CRISPR-based therapy PDUFA sets precedent for gene-editing commercialization timelines.

June 20, 2026 · NDA/BLA

Related catalysts(2)

Topline Data

— Casgevy

July 2, 2026 · N/A

PDUFA Date

Vertex Pharmaceuticals — Casgevy

First CRISPR-based therapy PDUFA sets precedent for gene-editing commercialization timelines.

June 20, 2026 · NDA/BLA

Indications

Sickle cell disease →

Therapeutic areas

Gene Therapy →
Hematology →
Hematology · Gene Therapy →

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