Table 1.
Ongoing gene therapy clinical trials for neurodegenerative disorders.
| Disorders | Trial code | Delivery route | Gene therapy | Phase |
|---|---|---|---|---|
| Alzheimer's disease | NCT00876863 | Direct basal forebrain injection | AAV2-NGF | Phase Ⅱ |
| Huntington's disease | NCT02519036 | Intrathecal injection | ASOs to HTT messenger RNA | Phase Ⅲ |
| Huntington's disease |
NCT03225833 NCT03225846 |
Intrathecal injection | ASOs to HTT mutant pre-messenger RNA | Phase Ⅰ |
| Pompe's disease | NCT02240407 | Intramuscular injection | AAV9-GAA | No results |
| Pompe's disease | NCT00976352 | Intramuscular injection | AAV1-GAA | Phase Ⅱ |
| Parkinson's disease |
NCT03065192 NCT01793543 |
Intraputaminal injection | AAV2-AADC | Phase Ⅰ |
| Parkinson's disease | NCT01621581 | Intraputaminal injection | AAV2-GDNF | Phase Ⅰ |
| Parkinson's disease | NCT02418598 | Intraputaminal injection | AAV2-AADC | Phase Ⅱ |
| Parkinson's disease |
NCT00400634 NCT00985517 |
Intraputaminal injection | AAV2-neurturin | Phase Ⅱ |
| Parkinson's disease | NCT00627588 | Intraputaminal injection | Lentivirus-AADC | Phase Ⅰ |
| Parkinson's disease | NCT00643890 | Injection into the subthalamic nucleus | AAV2-GAD | Phase Ⅱ |
| Metachromic leukodystrophy | NCT01801709 | Intracerebral injection | AAVrh10-ARSA | No results |
| Spinal muscular atrophy | NCT02122952 | Intravenous injection | AAV9-SMN | Phase Ⅰ |
| Spinal muscular atrophy | NCT02292537 | Intrathecal injection | ASOs targeting SMN2 splicing | Phase Ⅲ |
| Amyotrophic lateral sclerosis | NCT01041222 | Intrathecal injection | ASOs to SOD1 | Phase Ⅰ |
| Mucopolysaccharidosis type III A | NCT01474343 | Intracerebral injection | AAVrh10-SGSH | Phase Ⅰ |
| Mucopolysaccharidosis type III A | NCT02053064 | Intracerebral injection | AAVrh10-SUMF1 | Phase Ⅱ |
| Mucopolysaccharidosis type II | NCT03041324 | Intravenous injection | AAV6-IDS | Phase Ⅱ |
| Batten | NCT02725580 | Intrathecal injection | AAV9-CLN6 | Phase Ⅰ/Ⅱ |
| Batten | NCT01414985 | Intracranial injection | AAVrh10-TPP1 | Phase Ⅰ/Ⅱ |
| Batten | NCT01161576 | Intracranial injection | AAVrh10-TPP1 | Phase Ⅰ |
| Canavan | NA | Intraparenchymal injection | AAV2-ASPA | Phase Ⅰ |
NA, not applicable.