Targeted Genetics preclinical data

TGEN researchers and colleagues presented data showing that a single AAV vector encoding Factor VIII lacking the B-domain of the protein produced stable expression of the protein in the livers of mice for 12 months at levels equivalent to 20-27 percent of that normally seen in humans.

Data were presented at the American Society of Hematology meeting in New Orleans. TGEN expects to submit an IND for human testing of the gene therapy in 2001. ...