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AAV8-F8
- Development stage
- Preclinical
- Lead developer
- Children's Hospital of Philadelphia
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV8-F8 is an adeno-associated virus serotype 8 (AAV8) vector-based gene therapy designed for the treatment of Hemophilia A. It carries a codon-optimized expression cassette for human coagulation Factor VIII (F8). The therapy aims to provide sustained expression of Factor VIII in the liver following a single intravenous administration, thereby reducing or eliminating the need for regular factor replacement therapy. Research conducted at the Children's Hospital of Philadelphia (CHOP) has focused on optimizing the size of the F8 expression cassette to improve packaging efficiency, virion homogeneity, and transgene expression levels, as oversized cassettes can lead to incomplete packaging and reduced efficacy.
- Other names
- AAV8-hFVIIIAAV-8-hFVIIIAAV 8-hFVIII
02
Targets
F8 (Coagulation Factor VIIIa)
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