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Betibeglogene Autotemcel

CHEMBL4594311 Phase 4 معتمد Gene
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

A gene therapy used to treat beta-thalassemia, a genetic blood disorder in which the body produces insufficient functional hemoglobin. It involves modifying the patient's own stem cells to include a functional copy of the beta-globin gene, allowing the production of healthy red blood cells. It is a one-time treatment with the potential to reduce or eliminate the need for lifelong blood transfusions.

المجالات العلاجية

Lymphoma

آلية العمل

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Pharmacokinetics (PK)

Pharmacodynamics (PD)

الآلية

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

HBD / HBA

- / -

No targets recorded

Target interaction data is not yet available for this drug.

No interactions recorded

Drug interaction data is not yet available for this compound.

No side effects recorded

Side effect data is not yet available for this drug.

الأسئلة الشائعة

A gene therapy used to treat beta-thalassemia, a genetic blood disorder in which the body produces insufficient functional hemoglobin. It involves modifying the patient's own stem cells to include a functional copy of the beta-globin gene, allowing the production of healthy red blood cells. It is a one-time treatment with the potential to reduce or eliminate the need for lifelong blood transfusions.

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Yes, Betibeglogene Autotemcel is an approved drug. It has reached clinical phase 4. It is classified as a Gene.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

  • ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4594311. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.

إخلاء المسؤولية الطبية

This content is for educational and informational purposes only. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider before making medication decisions.

Data sources: ChEMBL, PubChem, DailyMed.