Hemophilia A is a bleeding disorder that results from mutations in the F8 gene, which codes for the Factor VIII protein essential to the clotting process. Patients with hemophilia A produce too little Factor VIII or an ineffective version of the protein. Conventional treatment for severe cases is typically through regular intravenous infusions of recombinant or plasma concentrated Factor VIII.<unk>  is a gene therapy that uses an adeno-associated virus 5 (AAV5) that codes for human Factor VIII, together with a human liver-specific promoter that encourages translation in liver endothelial and sinusoidal cells, where Factor VIII is ordinarily synthesised. By transfecting a functional version of the F8 gene into liver cells, the patient would be able to produce sufficient amounts of biologically
Valoctocogene roxaparvovec
Alipogene tiparvovec
Enadenotucirev
Neo-Viagra-Y.R.
Onasemnogene abeparvovec
Belantamab mafodotin
Atoltivimab/maftivimab/odesivimab
Sacituzumab govitecan