Adeno-associated Virus - Clinical Trials

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.

Selected Clinical Trials using AAV-Based Vectors
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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