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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