Adeno-associated virus
Clinical trials
Columns: Gene | Route of administration | Phase | Subject number | Status
CFTR | Lung, via aerosol | I | 12 | Complete | CFTR | Lung, via aerosol | II | 38 | Complete | CFTR | Lung, via aerosol | II | 100 | Complete | FIX | Intramuscular | I | 9 | Complete | FIX | Hepatic artery | I | 6 | Ended | TNFR:Fc | Intraarticular | I | 1 | Ongoing | AAT | Intramuscular | I | 12 | Ongoing | RPE65 | Subretinal | I-II | Multiple | Several ongoing and complete | sFlt-1 | Subretinal | I-II | 24 | Ongoing | Sarcoglycan | Intramuscular | I | 10 | Ongoing | GAD65, GAD67 | Intracranial | I | 12 | Complete | AAC | Intracranial | I | 21 | Ongoing | CLN2 | Intracranial | I | 10 | Ongoing | NGF | Intracranial | I | 6 | Ongoing