Fidanacogene Elaparvovec
A gene therapy used to treat adults with moderately severe to severe hemophilia B (a hereditary bleeding disorder). It works by delivering a functional copy of the gene needed to produce clotting factor IX, potentially providing long-term bleeding control after a single treatment.
Mecanismo de Ação
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)
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.
Perguntas frequentes
A gene therapy used to treat adults with moderately severe to severe hemophilia B (a hereditary bleeding disorder). It works by delivering a functional copy of the gene needed to produce clotting factor IX, potentially providing long-term bleeding control after a single treatment.
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, Fidanacogene Elaparvovec is an approved drug. It has reached clinical phase 4. It is classified as a Gene.
References & Data Sources
- ChEMBL — European Bioinformatics Institute (EBI). CHEMBL3990034. Open-access bioactivity database.
Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.
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