Table 1 Summary of results in naive... | American Society of Hematology

Table 1

Summary of results in naive hemophilia B dogs after ATVRX delivery of an AAV2-cFIX vector

Group/dog ID	Age at time of injection, mo	Sex	Weight, kg	Circulating cFIX, ng/mL*	Inhibitor (Bethesda unit)	Bleeding episodes (expected†)	Follow-up, mo
Low dose, 1 × 10¹² vg/kg
I04	6	M	15.5	32 ± 7	No	0 (28)	61
I05	9	M	17.5	141 ± 18	No	1 (27)	59
J04	4.5	M	12.4	27 ± 4	No	1 (19)	42
Mid dose, 3 × 10¹² vg/kg
H48	6.5	M	15	275 ± 75	No	0 (20)	44
H34	11	F	15	76 ± 14	No	1 (29)	63
I07	8	F	16	125 ± 39	No	0 (22)	47
High dose, 8.5 × 10¹² vg/kg
H24	9	M	24	20 ± 3	No	3 (29)	64
M25	3.5	F	8.7	23 ± 3	No	1 (6)	12
Mid dose, no immunosuppression
J03	11	M	17.5	81 ± 13	No	1 (20)	44
J62	6	M	14	120‡	Transient (1.5 BU)	6§ (5)	40

Group/dog ID	Age at time of injection, mo	Sex	Weight, kg	Circulating cFIX, ng/mL*	Inhibitor (Bethesda unit)	Bleeding episodes (expected†)	Follow-up, mo
Low dose, 1 × 10¹² vg/kg
I04	6	M	15.5	32 ± 7	No	0 (28)	61
I05	9	M	17.5	141 ± 18	No	1 (27)	59
J04	4.5	M	12.4	27 ± 4	No	1 (19)	42
Mid dose, 3 × 10¹² vg/kg
H48	6.5	M	15	275 ± 75	No	0 (20)	44
H34	11	F	15	76 ± 14	No	1 (29)	63
I07	8	F	16	125 ± 39	No	0 (22)	47
High dose, 8.5 × 10¹² vg/kg
H24	9	M	24	20 ± 3	No	3 (29)	64
M25	3.5	F	8.7	23 ± 3	No	1 (6)	12
Mid dose, no immunosuppression
J03	11	M	17.5	81 ± 13	No	1 (20)	44
J62	6	M	14	120‡	Transient (1.5 BU)	6§ (5)	40

Average ± SD of plateau levels > 3 months.

Based on an average of ∼ 5 bleeding episodes per year for untreated dogs.³⁷

Measured at day > 600 after resolution of inhibitor.

Occurred from days 15 to 363, while inhibitor persisted.