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Betibeglogene Autotemcel

CHEMBL4594311 Phase 4 Onaylandı 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.

Terapötik Alanlar

Lymphoma

Etki Mekanizması

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)

Mekanizma

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.

Sıkça Sorulan Sorular

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.

Tıbbi Sorumluluk Reddi

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.