Table 2.

Treatment-related adverse events

System organ class Preferred term	MPSa II				FIXb		ZFNcin vivo editing Overalld(N = 13) n (%) [T]
	Cohort 1 Dose level 5e12 vg/kg (N = 2) n (%) [T]e	Cohort 2 Dose level 1e13 vg/kg (N = 2) n (%) [T]	Cohort 3 Dose level 5e13 vg/kg (N = 5) n (%) [T]	Overall (N = 9) n (%) [T]	Cohort 3 Dose level 5e12 vg/kg (N = 1) n (%) [T]	Overall (N = 1) n (%) [T]
Any event	2 (100) [5]	1 (50.0) [6]	4 (80.0) [22]	7 (77.8) [33]	1 (50.0) [5]	1 (11.1) [5]	8(61.5) [38]
Cardiac disorders	0 (0.0) [0]	0 (0.0) [0]	0 (0.0) [0]	0 (0.0) [0]	1 (50.0) [1]	1 (11.1) [1]	1(7.7) [1]
Sinus tachycardia	0 (0.0) [0]	0 (0.0) [0]	0 (0.0) [0]	0 (0.0) [0]	1 (50.0) [1]	1 (11.1) [1]	1(7.7) [1]
Gastrointestinal disorders	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Pancreatic cyst	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
General disorders and administration site conditions	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	1 (50.0) [2]	1 (11.1) [2]	2(15.4) [3]
Chills	0 (0.0) [0]	0 (0.0) [0]	0 (0.0) [0]	0 (0.0) [0]	1 (50.0) [1]	1 (11.1) [1]	1(7.7) [1]
Pyrexia	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	1 (50.0) [1]	1 (11.1) [1]	2(15.4) [2]
Hepatobiliary disorders	0 (0.0) [0]	1 (50.0) [1]	2 (40.0) [3]	3 (33.3) [4]	0 (0.0) [0]	0 (0.0) [0]	3(23.1) [4]
Cholelithiasis	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Hepatic lesion	0 (0.0) [0]	1 (50.0) [1]	0 (0.0) [0]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Hepatomegaly	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Portal fibrosis	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Injury, poisoning, and procedural complications	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Infusion-related reaction	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Investigations	0 (0.0) [0]	1 (50.0) [2]	3 (60.0) [11]	4 (44.4) [13]	0 (0.0) [0]	0 (0.0) [0]	4(30.8) [13]
Alanine aminotransferase increased	0 (0.0) [0]	1 (50.0) [1]	2 (40.0) [6]	3 (33.3) [7]	0 (0.0) [0]	0 (0.0) [0]	3(23.1) [7]
Aspartate aminotransferase increased	0 (0.0) [0]	1 (50.0) [1]	2 (40.0) [3]	3 (33.3) [4]	0 (0.0) [0]	0 (0.0) [0]	3(23.1) [4]
Blood pressure systolic increased	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [2]	1 (11.1) [2]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [2]
Musculoskeletal and connective tissue disorders	1 (50.0) [1]	0 (0.0) [0]	0 (0.0) [0]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Muscular weakness	1 (50.0) [1]	0 (0.0) [0]	0 (0.0) [0]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Nervous system disorders	1 (50.0) [1]	0 (0.0) [0]	1 (20.0) [2]	2 (22.2) [3]	1 (50.0) [1]	1 (11.1) [1]	3(23.1) [4]
Dizziness	1 (50.0) [1]	0 (0.0) [0]	0 (0.0) [0]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Dysgeusia	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Headache	0 (0.0) [0]	0 (0.0) [0]	1 (20.0) [1]	1 (11.1) [1]	1 (50.0) [1]	1 (11.1) [1]	2(15.4) [2]
Skin and subcutaneous tissue disorders	2 (100) [3]	1 (50.0) [2]	1 (20.0) [1]	4 (44.4) [6]	0 (0.0) [0]	0 (0.0) [0]	4(30.8) [6]
Cold sweat	1 (50.0) [1]	0 (0.0) [0]	0 (0.0) [0]	1 (11.1) [1]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [1]
Erythema	0 (0.0) [0]	1 (50.0) [2]	0 (0.0) [0]	1 (11.1) [2]	0 (0.0) [0]	0 (0.0) [0]	1(7.7) [2]
Pruritus	1 (50.0) [2]	0 (0.0) [0]	1 (20.0) [1]	2 (22.2) [3]	0 (0.0) [0]	0 (0.0) [0]	2(15.4) [3]
Vascular disorders	0 (0.0) [0]	1 (50.0) [1]	2 (40.0) [2]	3 (33.3) [3]	1 (50.0) [1]	1 (11.1) [1]	4(30.8) [4]
Flushing	0 (0.0) [0]	1 (50.0) [1]	2 (40.0) [2]	3 (33.3) [3]	1 (50.0) [1]	1 (11.1) [1]	4(30.8) [4]

^aMPS, mucopolysaccharidosis.

^bFIX, factor IX; study SB-FIX-1501 did not include the 5e12 and 1e13 vg/kg doses, so it did not have cohorts 1 or 2, only cohort 3.

^cZFN, zinc-finger nuclease.

^dStudy SB-318-1502 did not report any treatment-related adverse events.

^eN, total number of subjects in each group; n, number of subjects at each SOC; [T], total number of adverse events. Each subject was counted only once for each applicable specific adverse event. A subject with multiple adverse events within a system organ class was counted only once for that system organ class. The table is sorted by alphabetical order.