Skip to content

Summary of clinical trials using AAV gene transfer

Disease Transgene product Serotype Route of administration Clinical trial ClinicalTrials.gov identifier Refs
AAV clinical trials for inherited diseases
α1 antitrypsin deficiency α1 antitrypsin AAV2 Intramuscular Phase I/II NCT00377416 101,102
AAV1 NCT00430768
Batten’s disease CLN2 AAV2 Direct intracranial administration Phase I NCT00151216 90
AAVrh10 NCT01161576
Canavan’s disease Aspartoacylase AAV2 Direct intracranial administration Phase I NA 89
Cystic fibrosis CFTR AAV2 Direct instillation to maxillary sinus, bronchoscopy to right lower lobe, aerosol to whole lung Phase I/II NCT00004533 154,155,156,157,158
Haemophilia B Factor IX AAV2 Intramuscular Phase I/II NCT00076557 36,39
Hepatic NCT00515710
AAV8 Intravenous NCT00979238
Leber’s congenital amaurosis RPE65 AAV2 Subretinal Phase I/II NCT00643747 4, 7, 17
NCT00516477
NCT00481546
LPL deficiency LPL AAV1 Intramuscular Phase I/II NCT01109498, NCT00891306 12,103,116
Pompe’s disease GAA AAV1 Series of intradiaphragmatic injections Phase I/II NCT00976352 NA (unpublished)
Muscular dystrophy: Duchenne Microdystrophin AAV1–AAV2 hybrid Intramuscular Phase I NCT00428935 97
Muscular dystrophy: limb girdle α-sarcoglycan AAV1 Two to six separate injections into the selected muscle Phase I NCT00494195 95,96
AAV clinical trials for acquired diseases
Severe heart failure SERCA2a AAV1 Antegrade epicardial coronary artery infusion Phase I/II NCT00454818 159
AAV6 NCT00534703
Parkinson’s disease AADC AAV2 Intracranial Phase I/II NCT00229736 64,65
GAD NCT00643890, NCT00195143, NCT01301573 66,69
Neutrophin NCT00252850, NCT00985517, NCT00400634 67,68
Age-related macular degeneration sFLT01 AAV2 Intravitreal injection Phase I NCT01024998 NA (unpublished)
Rheumatoid arthritis TNFR-Fc AAV2 Intra-articular Phase I NCT00617032, NCT00126724 160,161,162
AADC, aromatic-L-amino-acid decarboxylase; AAV, adeno-associated virus; CFTR, cystic fibrosis transmembrane regulator; CLN2, also known as tripeptidyl peptidase 1 (TPP1); GAA, acid α-glucosidase; GAD, glutamic acid decarboxylase; LPL, lipoprotein lipase; NA, not available; SERCA2a, sarcoplasmic reticulum calcium ATPase 2a; sFLT01, portion of the vascular endothelial growth factor natural receptor; TNFR-Fc, tumour necrosis factor receptor-immunoglobulin Fc fragment fusion protein.
Adeno-associated Virus (AAV) | signagen |

Leave a Reply

Your email address will not be published. Required fields are marked *