Table 3 Quantification of SPVN06 DNA in NHP tissue at Weeks 4 and 13 after SPVN06 subretinal bilateral injection.

From: Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies

SPVN06 DNA (copies/µg DNA)

Week 4

Week 13

6.0 × 1010 vg/eye

6.0 × 109 vg/eye

2.0 × 1010 vg/eye

6.0 × 1010 vg/eye

BLDa

BLQb

Mean Copyc

BLDa

BLQb

Mean Copyc

BLDa

BLQb

Mean Copyc

BLDa

BLQb

Mean Copyc

Ocular Tissue

 Retina left bleb

  

5 (7.87 × 106)

  

5 (8.24 × 105)

  

6 (2.17 × 106)

  

6 (5.02 × 106)

 Retina left remaining

  

6 (1.26 × 106)

  

5 (1.69 × 105)

  

6 (4.78 × 105)

  

6 (1.79 × 106)

 Aqueous humor

4

1

 

5

  

6

  

6

  

 Vitreous humor

  

5 (1.11 × 102)

4

1

 

3

2

1 (1.32 × 101)

3

2

1 (1.63 × 101)

 Bulbar and palpebral conjunctiva

1

 

4 (1.48 × 103)

4

1

 

3

 

3 (3.02 × 103)

 

1

5 (2.52 × 103)

 Choroid/RPE bleb

  

5 (1.10 × 107)

  

5 (1.32 × 106)

  

6 (3.91 × 106)

  

6 (7.46 × 106)

 Choroid/RPE remaining

  

5 (1.85 × 106)

  

5 (2.10 × 105)

  

6 (1.25 × 106)

  

6 (2.39 × 106)

 Cornea

  

5 (1.44 × 104)

 

3

2 (4.05 × 102)

  

6 (7.45 × 103)

  

6 (5.76 × 103)

 Iris/Ciliary body

  

5 (1.68 × 105)

  

5 (5.73 × 103)

  

6 (8.17 × 103)

  

6 (1.09 × 104)

 Lensd

  

5 (4.15 × 104)

 

1

4 (1.19 × 104)

 

1

5 (2.39 × 105)

  

6 (3.11 × 104)

 Sclera

  

5 (3.86 × 105)

  

5 (2.83 × 104)

  

6 (4.43 × 105)

  

6 (4.84 × 105)

 Optic nerve

  

5 (4.51 × 104)

  

5 (1.02 × 103)

  

6 (3.83 × 103)

  

6 (3.29 × 104)

Optic chiasm

  

6 (1.07 × 104)

 

1

4 (1.02 × 103)

  

6 (4.91 × 102)

  

6 (1.36 × 103)

Optic tract

  

6 (4.02 × 103)

 

1

5 (1.81 × 103)

  

6 (8.07 × 102)

  

6 (2.32 × 103)

Visual cortex

6

  

6

  

6

  

6

  

LGN

  

6 (7.66 × 102)

2

 

4 (1.16 × 103)

 

1

5 (2.69 × 102)

  

6 (5.95 × 102)

Cerebellum

6

  

6

  

6

  

6

  

Superior colliculus

1

3

2 (9.98 × 101)

2

3

1 (5.63 × 102)

2

1

3 (1.66 × 102)

 

2

4 (1.67 × 102)

Frontal cortex

3

3

 

6

  

6

  

4

2

 

Mandibular lymph nodes

 

1

5 (3.91 × 104)

2

2

2 (5.23 × 102)

 

2

4 (3.73 × 103)

 

1

5 (3.39 × 104)

Spleen

  

6 (2.18 × 104)

 

2

4 (5.33 × 102)

  

6 (2.24 × 103)

 

2

4 (6.12 × 103)

Heart

6

  

6

  

6

  

6

  

Kidney

5

 

1 (1.02 × 102)

6

  

6

  

6

  

Liver caudate

5

 

1 (7.39 × 102)

6

  

6

  

5

 

1 (1.23 × 102)

Liver lateral left

2

3

1 (7.19 × 102)

5

1

 

5

1

 

5

 

1 (1.68 × 102)

Liver lateral right

4

1

1 (8.19 × 102)

5

1

 

5

1

 

5

 

1 (1.38 × 102)

Liver median

3

2

1 (9.02 × 102)

5

1

 

5

1

 

5

 

1 (1.32 × 102)

Lung

4

 

2 (6.20 × 101)

6

  

6

  

6

  

Ovary

3

        

3

  

Testis

3

        

3

  
  1. aBLD was defined as <12.5 copies/µg of DNA.
  2. bBLQ was defined as <50 copies/µg of DNA.
  3. cQuantity (copies/µg) of DNA for tissue samples and copy/µl for aqueous and vitreous humors.
  4. dFor lens samples, quantification of SPVN06 DNA (vector genome) was reported as copies/samples.
  5. BLD below limit of detection, BLQ below lower limit of quantification, LGN lateral geniculate nucleus, NHP non-human primate, RPE retinal pigmented epithelium.
Back to article page