Table 3 Secondary and exploratory outcomes of SGCB expression in muscle biopsies (tibialis anterior and biceps) at Day 60 and Year 2

From: Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results

 

Cohort 1 (dose, 1.85 × 1013 vg kg−1)a (n = 3)

Cohort 2 (dose, 7.41 × 1013 vg kg−1)b (n = 3)c

Timepoint

Immunoblot (% NC)

Percentage SGCB+ fibers (% NC)

SGCB intensity (% NC)

Vector copies per nucleus

Immunoblot (% NC)

Percentage SGCB+ fibers (% NC)

SGCB intensity (% NC)

Vector copies per nucleus

 

qPCR

ddPCR

 

qPCR

ddPCR

Baseline mean (s.d.)

NA

8.9 (4.7)

7.4 (6.0)

0

NA

33.1 (4.8)

5.9 (6.4)

4.6 (5.7)

0

0

Median (range)

NA

8.5 (4.4, 13.7)

4.7 (3.3, 14.3)

0

NA

35.0 (27.6, 36.6)

5.0 (0, 12.8)

2.5 (0.2, 11.1)

0

0

Day 60 mean (s.d.)

36.2 (2.7)

51.0 (10.6)

47.4 (9.5)

0.6 (0.4)

NA

62.1 (8.7)

72.3 (6.2)

73.1 (21.8)

4.2 (2.8)

2.3 (0.9)

Median (range)

34.7 (34.6, 39.3)

48.9 (41.6, 62.5)

47.4 (38.0, 56.9)

0.4 (0.3, 1.1)

NA

63.1 (53.0, 70.3)

74.8 (65.3, 77.0)

67.1 (54.9, 97.3)

4.0 (1.6, 7.2)

2.4 (1.3, 3.1)

Year 2 mean (s.d.)

54.0 (16.1)

46.8 (21.3)

35.1 (22.9)

0.1 (0.1)

0.46 (0.36)

60.3 (21.4)

63.1 (21.6)

43.5 (33.2)

0.2 (0.1)

0.5 (0.3)

Median (range)

56.2 (37.0, 69.0)

58.6 (22.3, 59.7)

45.9 (8.8, 50.5)

0.1 (0.1, 0.2)

0.38 (0.2, 0.9)

60.3 (45.1, 75.4)

63.1 (47.9, 78.4)

43.5 (20.0, 67.0)

0.2 (0.2, 0.3)

0.5 (0.3, 0.8)

  1. Values are mean (s.d.) and median (range); NC, normal control.
  2. a1.85 × 1013 vg kg−1 (linear standard qPCR).
  3. b7.41 × 1013 vg kg−1 (linear standard qPCR).
  4. cn = 2 at Year 2.
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