Table 2.
Concentrations of the transgene product (KH631 protein) in different tissues of rhesus monkeys after a single subretinal injection of KH631 in bilateral eyes (ng/g for solid tissues or ng/mL for liquid tissues)
| Name | 4 wk postdose |
8 wk postdose |
13 wk postdose |
|||
|---|---|---|---|---|---|---|
| 1F001 | 1M001 | 1F002 | 1M002 | 1F003 | 1M003 | |
| Cornea | BLQ | BLQ | BLQ | 28.6 | 45.8 | 60.6 |
| Aqueous humor∗ | 118 | BLQ | 198 | 34.5 | 87.7 | 188 |
| Iris/Ciliary body | 415 | BLQ | 369 | 96.1 | 314 | 144 |
| Lens | BLQ | BLQ | BLQ | BLQ | 82.8 | 50.7 |
| Vitreous body∗ | 651 | 190 | 1930 | 253 | 580 | 1190 |
| Retina | 15400 | 26800 | 22400 | 10400 | 17000 | 13000 |
| Choroid | 9370 | 4330 | 7330 | 16400 | 9910 | 3850 |
| Sclera | 642 | 255 | BLQ | 103 | 769 | 178 |
| Optic nerve | BLQ | BLQ | BLQ | 104 | 65.4 | 34.5 |
| Conjunctiva | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Liver | 25.9 | 23.7 | 34.2 | 60.6 | 280 | 224 |
| Spleen | BLQ | BLQ | BLQ | BLQ | 42.4 | 32.8 |
| Feces | BLQ | BLQ | BLQ | BLQ | 34.2 | 55.7 |
| Preauricular Lymph nodes | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Lacrima | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Bone marrow∗ | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Cerebrum | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Forebrain | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Thalamus | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Jejunum | BLQ | BLQ | BLQ | BLQ | 36.1 | 49.9 |
| Kidney | BLQ | 71.4 | 64.8 | 106 | 349 | 318 |
| Nasal mucous | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Oral mucosa | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Skeletal muscle | BLQ | BLQ | BLQ | BLQ | 25.8 | 45.0 |
| Stomach | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Pancreas | BLQ | BLQ | BLQ | BLQ | 175 | 189 |
| Heart | BLQ | BLQ | BLQ | BLQ | 83.4 | 62.4 |
| Ovary | BLQ | – | BLQ | – | BLQ | – |
| Uterus | BLQ | – | BLQ | – | BLQ | – |
| Epididymis | – | BLQ | – | BLQ | – | BLQ |
| Prostate gland/Seminal vesicle | – | BLQ | – | BLQ | – | BLQ |
| Testis | – | BLQ | – | BLQ | – | BLQ |
| Lung | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
| Urine∗ | BLQ | BLQ | BLQ | BLQ | BLQ | BLQ |
“∗” means liquid tissues, with the unit of ng/mL; “–” means no tissue; BLQ means below the lower limit of quantification.