Technology evaluation: AAV factor IX gene therapy, Avigen Inc.

S A Fabb; J G Dickson

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PubMed · 11249763

Technology evaluation: AAV factor IX gene therapy, Avigen Inc.

S A Fabb·J G Dickson

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Abstract

Avigen has developed a recombinant adeno-associated viral vector expressing human blood-coagulation Factor IX (F.IX) for the potential treatment of hemophilia B. In a phase I clinical trial being conducted at The Children's Hospital of Philadelphia and Stanford University Medical Center, the vector, AAV-CMV-hF.IX (Coagulin-B), was injected at a low dose into three patients with severe hemophilia B. No evidence of toxicity, germline transmission of vector sequences, or formation of inhibitory antibodies against F.IX was observed, and in two of the three patients there was an indication of a modest clinical response.

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Animals Antibody Formation Biotechnology Dependovirus Factor IX Genetic Therapy Genetic Vectors Hemophilia B Humans Male Clinical Trial Clinical Trial, Phase I Journal Article Multicenter Study Review

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S A Fabb, J G Dickson. 2000. Technology evaluation: AAV factor IX gene therapy, Avigen Inc.. https://pubmed.ncbi.nlm.nih.gov/11249763/

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