Exagamglogene Autotemcel

Summary

Exagamglogene Autotemcel (CHEMBL5095474) is an approved gene (ATC B06AX05); indicated across 4 conditions including sickle cell disease and beta thalassemia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Gene
• ATC class: B06AX05
• Indications: 4 conditions
• Clinical trials: 7

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5095474
Name	Exagamglogene Autotemcel
Type	Gene
Max phase	4
ATC	B06AX05

Also known as: Casgevy, CTX-001, CTX001, Exagamglogene autotemcel, EXAGAMGLOGENE AUTOTEMCEL

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

4 indications (4 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
sickle cell disease	4	MONDO:0011382	MONDO:0011382
beta thalassemia	4	MONDO:0019402	Orphanet:848
thalassemia	4	MONDO:0000984	EFO:1001996

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE3	4
PHASE2/PHASE3	2
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04208529	PHASE3	ENROLLING_BY_INVITATION	A Long-term Follow-up Study in Participants Who Received CTX001
NCT05329649	PHASE3	ACTIVE_NOT_RECRUITING	Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
NCT05356195	PHASE3	ACTIVE_NOT_RECRUITING	Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
NCT05477563	PHASE3	RECRUITING	Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
NCT03655678	PHASE2/PHASE3	COMPLETED	A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
NCT03745287	PHASE2/PHASE3	COMPLETED	A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
NCT07392255	PHASE1/PHASE2	RECRUITING	An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).