Betibeglogene Autotemcel

Summary

Betibeglogene Autotemcel (CHEMBL4594311) is an approved gene (ATC B06AX02); indicated across 3 conditions including beta thalassemia and sickle cell disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Gene
• ATC class: B06AX02
• Indications: 3 conditions

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4594311
Name	Betibeglogene Autotemcel
Type	Gene
Max phase	4
ATC	B06AX02

Also known as: BB-305, Betibeglogen autotemcel, Betibeglogene autotemcel, LentiGlobin BB305, Zynteglo, BETIBEGLOGENE 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

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
beta thalassemia	4	MONDO:0019402	Orphanet:848

1 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
sickle cell disease	1	MONDO:0011382	MONDO:0011382

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

Clinical trials

Total trials: 0.

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).