|
Glycogen storage disease 1A |
Ultragenyx |
I/II |
R |
AAV8 |
NCT035117085 |
|
Crigler-Najjar |
Genethon-Selecta Bio |
I/II |
R |
AAV |
NCT03466463 |
|
Ornithine transcarbamylase deficiency |
University of Pennsylvania |
I |
T |
Adeno- viral |
NCT00004498 |
|
|
Ultragenyx |
I/II |
R |
AAV8 |
NCT02991144 |
Intermediary metabolism |
Methylmalonic acidaemia |
Moderna Therapeutics |
I/II |
R |
Non viral |
NCT03810690 |
|
Propionic acidaemia |
Moderna Therapeutics |
I/II |
A-NR |
Non viral |
NCT04159103 |
|
Phenylketonuria |
Homology Medicines |
I/II |
R |
AAVH- SC15 |
NCT03952156 |
|
|
National Taiwan University Hospital |
I/II |
T |
AAV2 |
NCT01395641 |
|
Aromatic L-amino acid decarboxylase deficiency |
National Taiwan University Hospital |
II |
R |
AAV2 |
NCT02926066 |
|
|
National Institute of Health |
I |
R |
AAV2 |
NCT02852213 |
Lipid metabolism |
Homozygous Familial Hypercholesterolaemia |
RegenX Bio |
I/II |
R |
AAV |
NCT02651675 |
|
|
Sangamo Therapeutics |
I/II |
H |
AAV6 |
NCT02702115 |
|
Mucopolysaccharidosis 1 |
RegenX Bio |
I |
R |
AAV9 |
NCT03580083 |
|
|
Orchard Therapeutics/San Raffaele-Telethon Institute for Gene Therapy |
I/II |
R |
LV |
NCT03488394 |
|
Mucopolysaccharidosis 2 |
Sangamo Therapeutics |
I/II |
H |
AAV6 |
NCT03041324 |
|
|
RegenX Bio |
I/II |
R |
AAV9 |
NCT03566043 |
|
|
Manchester University |
I/II |
R |
LV |
NCT04201405 |
|
|
Lysogene |
I/II |
T |
AAVrh10 |
NCT01474343 |
|
Mucopolysaccharidosis 3A |
Lysogene |
I/II |
R |
AAVrh10 |
NCT03612869 |
|
|
Abeona Therapeutics |
I/II |
R |
AAV9 |
NCT02716246; NCT04088734 |
|
Mucopolysaccharidosis 3B |
Abeona Therapeutics |
I/II |
R |
AAV9 |
NTC03315182 |
|
|
Uniqure |
I/II |
T |
AAV5 |
NCT03300453 |
|
Mucopolysaccharidosis 6 |
Fondazione Telethon |
I/II |
R |
AAV8 |
NCT03173521 |
|
|
Audentes Therapeutics |
I/II |
A-NR |
AAV8 |
NCT04174105 |
|
|
Spark Therapeutics |
I/II |
A-NR |
AAV |
NCT04093349 |
Lysosomal storage diseases |
Pompe disease |
Florida University |
I/II |
T |
AAV1 |
NCT00976352 |
|
|
Florida University |
I |
R |
AAV9 |
NCT02240407 |
|
Danon disease |
Rocket Pharmaceuticals |
I |
R |
AAV9 |
NCT03882437 |
|
|
Sangamo Therapeutics |
I/II |
R |
AAV6 |
NCT04046224 |
|
Fabry disease |
Freeline Therapeutics |
I/II |
R |
AAV |
NCT04040049 |
|
|
AvroBio |
I/II |
R |
LV |
NCT03454893 |
|
Ceroide lipofuscinosis 6 |
Amicus Therapeutics |
I/II |
A-NR |
AAV9 |
NCT02725580 |
|
Ceroide lipofuscinosis 3 |
Amicus Therapeutics |
I/II |
R |
AAV9 |
NCT03770572 |
|
|
Cornell University |
I |
A-NR |
AAV.rh10 |
NCT01161576 |
|
Ceroide lipofuscinosis 2 |
Cornell University |
I |
A-NR |
AAV2 |
NCT00151216 |
|
|
Cornell University |
I/II |
A-NR |
AAV.rh10 |
NCT01414985 |
|
GM1 Gangliosidosis |
National Human Genome Research Institute |
I/II |
R |
AAV9 |
NCT03952637 |
|
Metachromatic leukodystrophy |
Orchard Therapeutics/San Raffaele-Telethon Institute for Gene Therapy |
I/II |
R |
LV |
NTC03392987 |
|
|
Shenzhen University |
I/II |
R |
LV |
NCT02559830 |
|
Gaucher type 1 |
AvroBio |
I/II |
R |
LV |
NCT04145037 |
|
|
Bluebird Bio |
II/III |
A-NR |
LV |
NCT01896102 |
Peroxisomal disorders |
X-linked childhood cerebral adrenoleukodystrophy |
Bluebird Bio |
III |
A-NR |
LV |
NCT03852498 |
|
|
Shenzhen Second People's Hospital |
I/II |
R |
LV |
NCT0372755 |