Table 3.
The tissue tropisms of different AAV serotypes and the representative clinical trials.
| AAV Serotype | Tissue-Specific Tropisms | Key Pipeline | Disease | The Delivered Gene | Sponsor (s) | The Clinical Trial Stage |
|---|---|---|---|---|---|---|
| AAV1 | Muscle, heart, skeletal muscle (including cardiac muscle), nerve tissue | Glybera | Lipoprotein lipase deficiency | Lipoprotein lipase | UniQure | Approved # |
| AAV2 | Central nervous system, muscle, liver, brain tissue, eye | BIIB111 | Hereditary ophthalmopathy | Rab escort protein 1 | NightstaRx Ltd., a Biogen Company | Phase III completed, suspended |
| AAV3 | Muscles, liver, lung, eye | N/A | N/A | N/A | N/A | N/A |
| AAV4 | Central nervous system, muscle, eye, brain | N/A | N/A | N/A | N/A | N/A |
| AAV5 | Lung, eye, central nerve, joint synovium, pancreas | BMN 270 | Hemophilia type-A | Coagulation factor VIII | BioMarin Pharmaceutical | Approved |
| AAV6 | Lung, heart | SB-525 | Hemophilia type-A | Coagulation factor VIII | Pfizer | Phase III |
| AAV7 | Muscle, liver | N/A | N/A | N/A | N/A | N/A |
| AAV8 | Liver, eye, central nerve, muscle | BIIB112 | X-linked retinoschisis | Pigmentosa GTPase regulator | NightstaRx Ltd., a Biogen Company | Phase III, suspended |
| AAV9 | Heart, muscle, lung(alveolar), liver, central nervous system | PF-06939926 | Duchenne muscular dystrophy | Truncated dystrophin | Pfizer | Phase III |
| AAV-DJ | Liver, retina, lung, kidney | N/A | N/A | N/A | N/A | N/A |
| AAV-DJ/8 | Liver, eye, central nervous system, muscle | N/A | N/A | N/A | N/A | N/A |
| AAV-Rh10 | Lung, heart, muscle, central nervous system, liver | LYS-GM101 | GM1 gangliosidosis | beta-galactosidase | Lysogene | Phase II |
| AAV11 | Unknown | N/A | N/A | N/A | N/A | N/A |
| AAV12 | Nasal | N/A | N/A | N/A | N/A | N/A |
| AAV13 | Unknown | N/A | N/A | N/A | N/A | N/A |
N/A: Not available. #: Withdrawal of the marketing authorization.