Valoctocogene roxaparvovec: Difference between revisions
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Revision as of 21:04, 8 February 2025
Valoctocogene Roxaparvovec (also known as BMN 270) is an experimental gene therapy treatment for Hemophilia A. It is being developed by BioMarin Pharmaceutical Inc.
Etymology
The name "Valoctocogene Roxaparvovec" is derived from the components of the therapy. "Valo" refers to the valine amino acid used in the therapy, "octo" refers to factor VIII, which the therapy aims to increase, "gene" refers to the gene therapy aspect of the treatment, "roxaparvo" is derived from the adeno-associated virus serotype 5 (AAV5) used as a vector in the therapy, and "vec" is short for vector.
Mechanism of Action
Valoctocogene Roxaparvovec works by delivering a functional copy of the factor VIII gene to the patient's liver cells using an AAV5 vector. This allows the liver cells to produce a continuous supply of factor VIII, which is deficient in people with Hemophilia A.
Clinical Trials
As of 2021, Valoctocogene Roxaparvovec is in Phase 3 clinical trials. The therapy has shown promise in early trials, with patients experiencing increased levels of factor VIII and decreased bleeding episodes.
