. 2021 Feb 8;6:53. doi: 10.1038/s41392-021-00487-6

Table 2.

A selection of ongoing clinical trials employing AAV vectors

Condition	Intervention	Sponsor	Trial stage	Identifier
AADC deficiency	AADC	Krystof Bankiewicz UCSF	Phase I	NCT02852213
AADC deficiency	AADC	National Taiwan University Hospital	Phase II	NCT02926066
Batten disease (CLN2)	CLN2	Weill Cornell	Phase I/II	NCT01414985
Batten disease (CLN6)	CLN6	Nationwide Children’s Hospital	Phase I/II	NCT02725580
MPS-IIIB	NAGLU	UniQure	Phase I/II	NCT03300453
Parkinson disease	AADC	Jichi Medical University	Phase I/II	NCT02418598
	GDNF	NINDS	Phase I	NCT01621581
	Neurturin	Sangamo	Phase I/II	NCT00985517
	AADC	Voyager	Phase I	NCT03065192
SMA	SMN	AveXis	Phase III	NCT03461289
GAN	GAN	NINDS	Phase I	NCT02362438
Achromatopsia	CNGB3	AGTC	Phase I/II	NCT02599922
Achromatopsia	CNGB3	MeiraGTx	Phase I/II	NCT03001310
Choroideremia	REP1	Nightstar	Phase III	NCT03496012
	REP1	Spark	Phase I/II	NCT02341807
	REP1	STZ Eyetrial	Phase II	NCT02671539
	REP1	University of Oxford	Phase II	NCT02407678
LCA	RPE65	Spark	Phase III	NCT00999609
LCA	RPE65	MeiraGTx	Phase I/II	NCT02781480
LHON	ND4	GenSight	Phase III	NCT03293524
LHON	ND4	John Guy University of Miami	Phase I	NCT02161380
RP (RLBP1)	RLBP1	Novartis	Phase I/II	NCT03374657
Wet AMD	Anti-VEGF antibody	Regenxbio	Phase I	NCT03066258
Wet AMD	Anti-VEGF protein	Adverum Biotechnologies	Phase I	NCT03748784
X-Linked RP	RPGR	AGTC	Phase I/II	NCT03316560
	RPGR	MeiraGTx	Phase I/II	NCT03252847
	RPGR	Nightstar	Phase I/II	NCT03116113
X-linked retinoschisis	RS1	AGTC	Phase I/II	NCT02416622
X-linked retinoschisis	RS1	NEI	Phase I/II	NCT02317887
Crigler–Najjar syndrome	UGT1A1	Audentes	Phase I/II	NCT03223194
Crigler–Najjar syndrome	UGT1A1	Genethon	Phase I/II	NCT03466463
FH (homozygous)	LDLR	University of Pennsylvania	Phase I/II	NCT02651675
GSD1a	G6PC	Ultragenyx	Phase I/II	NCT03517085
Hemophilia A	FVIII	Shire	Phase I/II	NCT03370172
	FVIII	Bayer	Phase I/II	NCT03588299
	FVIII	BioMarin	Phase III	NCT03392974
	FVIII	Sangamo	Phase I/II	NCT03061201
	FVIII	Spark	Phase I/II	NCT03003533
	FVIII	UCL	Phase I	NCT03001830
Hemophilia B	FIX	Shire	Phase I/II	NCT01687608
	FIX	Pfizer	Phase II	NCT02484092
	FIX	Pfizer	Phase III	NCT03587116
	FIX	Sangamo	Phase I	NCT02695160
	FIX	St. Jude Children’s Research Hospital	Phase I	NCT00979238
	FIX	UniQure	Phase III	NCT03569891
	FIX	UCL	Phase I	NCT03369444
	FIX	Freeline Therapeutics	Phase II/III	NCT03641703
MPS-I	ZFN1, ZFN2, IDUA donor	Sangamo	Phase I	NCT02702115
MPS-II	ZFN1, ZFN2, IDS donor	Sangamo	Phase I	NCT03041324
MPS-IIIA	SGSH	LYSOGENE	Phase II/III	NCT03612869
MPS-VI	ARSB	Fondazione Telethon	Phase I/II	NCT03173521
OTC deficiency	OTC	Ultragenyx	Phase I/II	NCT02991144
A1AT deficiency	A1AT	UMMS	Phase I	NCT00377416
CMT1A	NTF3	Nationwide Children’s Hospital	Phase I/II	NCT03520751
DMD	Microdystrophin	Nationwide Children’s Hospital	Phase I/II	NCT03375164
	Mini-dystrophin	Pfizer	Phase I	NCT03362502
	Microdystrophin	Solid Biosciences	Phase I/II	NCT03368742
	Microdystrophin	Sarepta Therapeutics	Phase II	NCT03769116
LGMD, type 2E	LGMD2E	Sarepta Therapeutics	Phase I/II	NCT03652259
Dysferlinopathy	DYSF	Nationwide Children’s Hospital	Phase I	NCT02710500
HIV infections	PG9 antibody	International AIDS Vaccine Initiative	Phase I	NCT01937455
HIV infections	VRC07 antibody	NIAID	Phase I	NCT03374202
Pompe disease	GAA	Actus Therapeutics	Phase I/II	NCT03533673
Pompe disease	GAA	University of Florida	Phase I	NCT02240407
X-linked MTM	MTM1	Audentes	Phase I/II	NCT03199469

A1AT α1 antitrypsin, AADC aromatic l-amino acid decarboxylase, AGTC Applied Genetic Technologies Corporation, AMD age-related macular degeneration, ARSB arylsulfatase B, CLN2 neuronal ceroid lipofuscinosis type 2, CMT1A Charcot–Marie–Tooth disease type 1A, CNGB3 cyclic nucleotide-gated channel-β3, DMD Duchenne muscular dystrophy, DYSF dysferlin, FH familial hypercholesterolemia, FVIII factor VIII, G6PC glucose-6-phosphatase catalytic subunit, GAA α-glucosidase, GAN gigaxonin, GDNF glial cell line-derived neurotrophic factor, GSD1a glycogen storage disease type 1a, LCA Leber congenital amaurosis, LDLR low-density lipoprotein receptor, LHON Leber hereditary optic neuropathy, mAb monoclonal antibody, MPS mucopolysaccharidosis, MTM myotubular myopathy, NAGLU N-α-acetylglucosaminidase, ND not disclosed, ND4 NADH-ubiquinone oxidoreductase chain 4, NEI National Eye Institute, NIAID National Institute of Allergy and Infectious Diseases, NINDS National Institute of Neurological Disorders and Stroke, NTF3 neurotrophin 3, OTC ornithine transcarbamylase, REP1 RAB escort protein 1, RLBP1 retinaldehyde-binding protein 1, RP retinitis pigmentosa, RPE65 retinal pigment epithelium-specific 65 kDa protein, RPGR retinitis pigmentosa GTPase regulator, RS1 retinoschisin 1, SGSH N-sulfoglucosamine sulfohydrolase, SMA spinal muscular atrophy, SMN survival of motor neuron, UCL University College London, UCSF University of California San Francisco, UGT1A1 UDP glucuronosyltransferase family 1 member A1, UMMS University of Massachusetts Medical School, VEGF vascular endothelial growth factor, ZFN zinc-finger-containing protein.