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Onasemnogene Abeparvovec

CHEMBL4297240 Phase 4 Approuvé Gene
Half-Life
—
Bioavailability
—
Protein Binding
—
Molecular Weight
— g/mol
LogP
—
Phase
4

A one-time gene therapy that delivers a functional copy of the SMN1 gene to treat spinal muscular atrophy, a life-threatening genetic disease that causes progressive muscle weakness. By restoring the missing protein, it can dramatically improve motor function and survival in affected children.

Aires thérapeutiques

Non-Small Cell Lung Cancer

Mécanisme d'action

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)

Mécanisme

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.

Foire aux questions

A one-time gene therapy that delivers a functional copy of the SMN1 gene to treat spinal muscular atrophy, a life-threatening genetic disease that causes progressive muscle weakness. By restoring the missing protein, it can dramatically improve motor function and survival in affected children.

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, Onasemnogene Abeparvovec 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). CHEMBL4297240. Open-access bioactivity database.

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

Avertissement médical

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.