Clinical Trials
To date, AAV vectors have been used in over 80 clinical trials worldwide. Recently, promising results have been obtained from Phase 1 and Phase 2 trials for a number of diseases, including Leber's Congenital Amaurosis, Hemophilia, congestive heart failure, lipoprotein lipase deficiency (reference needed), and Parkinson's disease.
| Indication | Gene | Route of administration | Phase | Subject number | Status |
| Cystic fibrosis | CFTR | Lung, via aerosol | I | 12 | Complete |
| CFTR | Lung, via aerosol | II | 38 | Complete | |
| CFTR | Lung, via aerosol | II | 100 | Complete | |
| Hemophilia B | FIX | Intramuscular | I | 9 | Complete |
| FIX | Hepatic artery | I | 6 | Ended | |
| Arthritis | TNFR:Fc | Intraarticular | I | 1 | Ongoing |
| Hereditary emphysema | AAT | Intramuscular | I | 12 | Ongoing |
| Leber's Congenital Amaurosis | RPE65 | Subretinal | I-II | Multiple | Several ongoing and complete |
| Age-Related Macular Degeneration | sFlt-1 | Subretinal | I-II | 24 | Ongoing |
| Muscular dystrophy | Sarcoglycan | Intramuscular | I | 10 | Ongoing |
| Parkinson's | GAD65, GAD67 | Intracranial | I | 12 | Complete |
| Canavan's | AAC | Intracranial | I | 21 | Ongoing |
| Batten's | CLN2 | Intracranial | I | 10 | Ongoing |
| Alzheimer's | NGF | Intracranial | I | 6 | Ongoing |
Trials for the treatment of prostate cancer have reached phase III, however these ex vivo studies do not involve direct administration of AAV to patients.
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Famous quotes containing the word trials:
“All trials are trials for one’s life, just as all sentences are sentences of death.”
—Oscar Wilde (1854–1900)