Table 8. Bioactivity and efficacy... | American Society of Hematology

Table 8.

Bioactivity and efficacy studies in subjects treated with intramuscular AAV-hFIX

	PCR on muscle biopsy	Southern blot on muscle biopsy^8-150	F.IX immunohistochemistry	Max circ of F.IX	Decrease in F.IX infusion
A	Pos^8-151	Neg	Neg	1.40%	50%
B	Pos^8-151	ND	Pos	<1%	50%
C	Pos^8-151	Pos (4)	Neg	<1%	None
D	Pos	Pos (1.5)	Pos	<1%	None
	Pos	Pos (2.5)	Pos	<1%	None
E	Neg	Neg	Pos	<1%	None
F	Neg	Neg	Pos	1%	None
	Pos	Pos (0.5)	Pos	1%	None
G	Pos	Neg	Pos	<1%	None
H	Pos	Pos (0.5)	Pos	<1%	None

	PCR on muscle biopsy	Southern blot on muscle biopsy^8-150	F.IX immunohistochemistry	Max circ of F.IX	Decrease in F.IX infusion
A	Pos^8-151	Neg	Neg	1.40%	50%
B	Pos^8-151	ND	Pos	<1%	50%
C	Pos^8-151	Pos (4)	Neg	<1%	None
D	Pos	Pos (1.5)	Pos	<1%	None
	Pos	Pos (2.5)	Pos	<1%	None
E	Neg	Neg	Pos	<1%	None
F	Neg	Neg	Pos	1%	None
	Pos	Pos (0.5)	Pos	1%	None
G	Pos	Neg	Pos	<1%	None
H	Pos	Pos (0.5)	Pos	<1%	None

Max circ indicates maximum circulation of F.IX; ND, study not performed because of insufficient tissue.

Gene copy number estimates, based on comparison to standards, are shown in parentheses.

See Kay et al.24