Table 2. Design of the 9-month safety study in New Zealand White rabbits and the number of animals tested at each time point for any vector dose and vehicle.

Examinations	BaselineDose (vg/eye)					2nd weekDose (vg/eye)					3rd monthDose (vg/eye)					6th monthDose (vg/eye)					9th monthDose (vg/eye)
	2E9	2E10	2E11	1.5E12	Veh	2E9	2E10	2E11	1.5E12	Veh	2E9	2E10	2E11	1.5E12	Veh	2E9	2E10	2E11	1.5E12	Veh	2E9	2E10	2E11	1.5E12	Veh
In-life
Ophthalmic examinationa	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	12	12	12	11	13	6	6	6	6	7
IOP	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	12	12	12	11	13	6	6	6	6	7
ERG	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	N	N	N	N	N	6	6	6	6	7
Clinical pathology	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	12	12	12	12	13	6	6	6	6	7
Anti-AAV8 capsid Ab	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6	6
Anti-hRS Ab, hRS Prot.	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4
Vector biodistributionb	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	12	12	12	12	14	6	6	6	6	7
PBMC	N	N	N	N	N	6	6	6	6	7	6	6	6	6	7	6	6	6	6	7	6	6	6	6	7
Clinical observationc	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	12	12	12	12	14	6	6	6	6	7
Body weightc	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	12	12	12	12	14	6	6	6	6	7
Food consumptionc	24	24	24	24	28	24	24	24	24	28	18	18	18	18	21	12	12	12	12	14	6	6	6	6	7
Postmortem
Organ pathology	N	N	N	N	N	6	6	6	6	7	6	6	6	6	7	6	6	6	6	7	6	6	6	6	7
Ocular pathology	N	N	N	N	N	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4	4
Vector biodistributiond	N	N	N	N	N	2	2	2	2	3	2	2	2	2	3	2	2	2	2	3	2	2	2	2	3

ERG, electroretinogram; IOP, intraocular pressure; N, none; PBMC, peripheral blood mononuclear cell; Veh, vehicle.

^aOphthalmic examination was performed also at 2-day postinjection in all the rabbits.

^bAnalyzed in blood samples.

^cPerformed weekly for the entire 9-month period.

^dAnalyzed in vitreous humor samples.