{"id":137,"date":"2023-05-22T15:10:28","date_gmt":"2023-05-22T15:10:28","guid":{"rendered":"https:\/\/roctavianhcp-dev-001.azurewebsites.net\/en-us-new\/roctavian\/?page_id=137"},"modified":"2023-12-21T09:53:08","modified_gmt":"2023-12-21T09:53:08","slug":"how-roctavian-works","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/roctavian\/how-roctavian-works\/","title":{"rendered":"How ROCTAVIAN Works"},"content":{"rendered":"
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How ROCTAVIAN Works\n<\/h1>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n
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Creating the therapeutic vector\n<\/h4>\n \n

Functional Factor VIII genetic material is created in a lab and packaged into the empty viral shells of nonpathogenic adeno-associated virus serotype 5 (AAV5). The viral shells act as transport vehicles. This combination creates the therapeutic AAV5 vector, ROCTAVIAN.1,2<\/sup><\/p>\n <\/div>\n<\/div>\n\n

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Targeting the liver\n<\/h4>\n \n

The vectors are delivered into the bloodstream via a one-time intravenous infusion. Transcription of the genetic material is designed to occur within hepatocytes.1,3<\/sup><\/p>\n <\/div>\n<\/div>\n\n

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Forming episomes\n<\/h4>\n \n

Once inside the cells, functional genes are designed to form circular segments of DNA, called episomes, that exist on their own. These episomes are not designed to integrate into the host DNA. Low levels of vector integration have been found following ROCTAVIAN administration.1<\/sup><\/p>\n

The integration of liver-targeting AAV vector DNA into the genome may carry the theoretical risk of hepatocellular carcinoma development. ROCTAVIAN can also insert into the DNA of other human body cells. Monitor patients with risk factors for hepatocellular carcinoma with regular liver ultrasound and alpha-fetoprotein testing for 5 years following ROCTAVIAN administration.1<\/sup><\/p>\n <\/div>\n<\/div>\n\n

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Producing factor\n<\/h4>\n \n

Inside the cell nucleus, a liver-specific promoter instructs the cells to produce the Factor VIII protein.1<\/sup><\/p>\n <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

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