Table 2.
AAV clinical studies using immunosuppressants.
| Disease | Immune suppression/modulation | Trial ID | Drug name | Serotype |
|---|---|---|---|---|
| Achromatopsia | Corticosteroids | NCT02599922 | AGTC-401 | AAV2 |
| Achromatopsia | Corticosteroids | NCT02935517 | AGTC-402 | AAV2 |
| Achromatopsia | Corticosteroids | NCT02610582 | rAAV.hCNGA3 | AAV8 |
| Acute intermittent porphyria | Corticosteroids | NCT02082860 | rAAV2/5-PBGD | AAV5 |
| Alpha-1 antitrypsin deficiency | Corticosteroids | NCT02168686 | ADVM-043 | AAVrh10 |
| Amyotrophic lateral sclerosis | Prednisone, methylprednisolone | N/A | AAV-miR-SOD1 | AAVrh10 |
| Becker muscular dystrophy | Corticosteroids | NCT01519349 | AAV1-Follistatin | AAV1 |
| Canavan | Prednisone | NCT04833907 | rAAV-Olig001-ASPA | Undisclosed |
| Canavan | Rituximab, Sirolimus, Methylprednisolone | NCT05317780 | rAAV9-CB6-AspA | AAV9 |
| Choroideremia | Prednisolone | NCT01461213 | BIIB111 | AAV2 |
| Choroideremia | Prednisolone | NCT02077361 | BIIB111 | AAV2 |
| Choroideremia | Prednisolone/prednisone | NCT02407678 | BIIB111 | AAV2 |
| Choroideremia | Prednisolone/prednisone, dexmethasone, Moxifloxacin | NCT02671539 | BIIB111 | AAV2 |
| Danon | Sirolimus | NCT03882437 | RP-A501 | AAV9 |
| Diabetic macular edema | Corticosteroids | NCT04418427 | ADVM-022 | AAV2.7m8 |
| Duchenne muscular dystrophy | Corticosteroids | NCT00428935 | d3990 | AAV2.5 |
| Duchenne muscular dystrophy | Corticosteroids | 2020-002093-27 | GNT0004 | AAV8 |
| Duchenne muscular dystrophy | Corticosteroids | NCT04281485 | PF-06939926 | AAV9 |
| Duchenne muscular dystrophy | Corticosteroids | NCT02376816 | SRP-9001 | AAVrh74 |
| Duchenne muscular dystrophy | Corticosteroids | NCT03333590 | rAAVrh74.MCK. GALGT2 | AAVrh74 |
| Duchenne muscular dystrophy | Corticosteroids | NCT03375164 | SRP-9001 | AAVrh74 |
| Duchenne muscular dystrophy | Corticosteroids | NCT03769116 | SRP-9001 | AAVrh74 |
| Duchenne muscular dystrophy | Corticosteroids | NCT04626674 | SRP-9001 | AAVrh74 |
| Duchenne muscular dystrophy | Corticosteroids | NCT05096221 | SRP-9001 | AAVrh74 |
| Duchenne muscular dystrophy | Corticosteroids | NCT04240314 | AT702 | scAAV9 |
| Duchenne muscular dystrophy | Corticosteroids, eculizumab | NCT03362502 | PF-06939926 | AAV9 |
| Duchenne muscular dystrophy | Corticosteroids, eculizumab | NCT03368742 | SGT-001 | AAV9 |
| Duchenne muscular dystrophy | Prednisone | NCT02354781 | rAAV1.CMV. huFollistatin344 | AAV1 |
| Fabry | Corticosteroids | NCT04519749 | 4D-310 | 4D-A101 |
| Fabry | Corticosteroids | NCT04046224 | ST-920 | AAV2/6 |
| Fabry | Corticosteroids | NCT04040049 | FLT190 | AAV8 |
| Frontaltemporal dementia | Corticosteroids | NCT04747431 | PBFT02 | AAVhu68 |
| Frontaltemporal dementia | Methylprednisolone, Sirolimus, Prednisone, Rituximab | NCT04408625 | PR006 | AAV9 |
| Giant axonal neuropathy | Corticosteroids | NCT02362438 | TSHA-120 | scAAV9 |
| Glycogen storage disease | Corticosteroids | NCT03517085 | DTX401 | AAV8 |
| GM1 gangliosidosis | Corticosteroids | NCT04713475 | PBGM01 | AAVhu68 |
| GM1 gangliosidosis | Rituximab, Sirolimus, Methylprednisolone, Prednisone | NCT03952637 | AXO-AAV-GM1 | AAV9 |
| GM2 gangliosidosis | Corticosteroids | NCT04669535 | AXO-AAV-GM2 | AAVrh8 |
| Hemophilia A | Corticosteroids | NCT03003533 | SPK-8011 | AAV-LK03 |
| Hemophilia A | Corticosteroids | NCT03061201 | SB-525 | AAV2/6 |
| Hemophilia A | Corticosteroids | NCT03001830 | GO-8 | AAV2/8 |
| Hemophilia A | Corticosteroids | NCT02576795 | Roctavian | AAV5 |
| Hemophilia A | Corticosteroids | NCT03370913 | Roctavian | AAV5 |
| Hemophilia A | Corticosteroids | NCT03520712 | BMN 270 | AAV5 |
| Hemophilia A | Corticosteroids | NCT03392974 | Roctavian | AAV5 |
| Hemophilia A | Prednisolone | NCT03588299 | DTX201 | AAVhu37 |
| Hemophilia A | Prednisone, prednisolone | NCT03370172 | BAX 888 | AAV8 |
| Hemophilia B | Corticosteroids | NCT02484092 | SPK-9001 | AAV-Spark100 |
| Hemophilia B | Corticosteroids | NCT03307980 | SPK-9001 | AAV-Spark100 |
| Hemophilia B | Corticosteroids | NCT02695160 | SB-FIX | AAV2/6 |
| Hemophilia B | Corticosteroids | NCT03569891 | AMT-061 | AAV5 |
| Hemophilia B | Corticosteroids | NCT02396342 | AMT-060 | AAV5 |
| Hemophilia B | Corticosteroids | NCT03489291 | AMT-061 | AAV5 |
| Hemophilia B | Corticosteroids | NCT01687608 | AskBio009 (BAX 335) | AAV8 |
| Hemophilia B | Corticosteroids | NCT02618915 | DTX101 | AAVrh10 |
| Hemophilia B | Corticosteroids | NCT00979238 | scAAV2/8-LP1- hFIXco | scAAV2/8 |
| Hemophilia B | Corticosteroids, tacrolimus | NCT03369444 | FLT180a | AAVS3 |
| Hemophilia B | Prednisolone | NCT01620801 | SPK-9001 | AAV8 |
| Leber congenital amaurosis | Corticosteroids | NCT00643747 | tgAAG76 | AAV2 |
| Leber congenital amaurosis | Corticosteroids | NCT00516477 | Luxturna | AAV2 |
| Leber congenital amaurosis | Corticosteroids | NCT00481546 | rAAV2-CBSB-hRPE65 | AAV2 |
| Leber congenital amaurosis | Corticosteroids | NCT00999609 | Luxturna | AAV2 |
| Leber congenital amaurosis | Corticosteroids | NCT01208389 | Luxturna | AAV2 |
| Leber congenital amaurosis | Prednisone, Triamcinolone acetonide | NCT03920007 | SAR439483 | AAV5 |
| Limb-girdle muscular dystrophy | Corticosteroids | NCT05230459 | LION-101 | AAV9 |
| Limb-girdle muscular dystrophy | Prednisone | NCT03652259 | SRP-9003 | AAVrh74 |
| Leber hereditary optic neuropathy | Corticosteroids | NCT02652767 | GS010 | AAV2 |
| Leber hereditary optic neuropathy | Corticosteroids | NCT02161380 | scAAV2-P1ND4v2 | scAAV2tYF |
| Leber hereditary optic neuropathy | Corticosteroids, anti-inflammatory agent | NCT02064569 | GS010 | AAV2 |
| Lipoprotein lipase deficiency | Mycophenolate mofetil, cyclosporine | NCT01109498 | Glybera | AAV1 |
| Lipoprotein lipase deficiency | Mycophenolate mofetil, cyclosporine, methylprednisolone | NCT00891306 | Glybera | AAV1 |
| Lipoprotein lipase deficiency | Mycophenolate mofetil, cyclosporine, prednisolone | NCT02904772 | Glybera | AAV1 |
| Metachromatic leukodystrophy | Corticosteroids | NCT01801709 | AAVrh.10cuARSA | AAVrh10 |
| Methylmalonic acidemia | Corticosteroids | NCT04581785 | LB-001 | LK03 |
| Mucopolysaccharidosis type I | Corticosteroids | NCT02702115 | SB-318 | AAV2/6 |
| Mucopolysaccharidosis type II | Corticosteroids | NCT03566043 | RGX-121 | AAV9 |
| Mucopolysaccharidosis type II | Methyprednisolone, prednisone, sirolimus, tacrolimus | NCT05238324 | HMI-203 | AAVHSC |
| Mucopolysaccharidosis type II | Prednisone | NCT03041324 | SB-913 | AAV2/6 |
| Mucopolysaccharidosis type IIIA | Tacrolimus, mycophenolate mofetil, steroids | NCT03612869 | SAF-302 | AAVrh10 |
| Mucopolysaccharidosis type IIIB | Tacrolimus, mycophenolate mofetil | NCT03300453 | AMT110 | AAV2/5 |
| Mucopolysaccharidosis type VI | Corticosteroids | NCT03173521 | AAV2/8.TBG. hARSB | AAV2/8 |
| Neovascular age-related macular degeneration | Corticosteroids | NCT01024998 | AAV2-sFLT01 | AAV2 |
| Neovascular age-related macular degeneration | Corticosteroids | NCT03748784 | ADVM-022 | AAV2.7m8 |
| Neovascular age-related macular degeneration | Prednisolone | NCT03585556 | HMR59 | AAV2 |
| Ornithine transcarbamylase deficiency | Prednisone | NCT02991144 | DTX301 | scAAV8 |
| Parkinson’s disease | Methylprednisolone, Sirolimus, Prednisone, Rituximab | NCT04127578 | PR001 | AAV9 |
| Phenylketonuria | Corticosteroids | NCT03952156 | HMI-102 | AAVHSC15 |
| Pompe | Corticosteroids | NCT04174105 | AT845 | AAV8 |
| Pompe | Prednisone | NCT03533673 | ACTUS-101 | AAV2/8 |
| Pompe | Rituximab, Sirolimus | NCT02240407 | rAAV9-DES-hGAA | AAV9 |
| Pompe | Rituximab, Sirolimus, Corticosteroids | NCT00976352 | rAAV1-CMV-GAA | AAV1 |
| Spinal muscular atrophy | Corticosteroids, eculizumab | NCT03381729 | Zolgensma | scAAV9 |
| Spinal muscular atrophy | Corticosteroids, eculizumab | NCT02122952 | Zolgensma | scAAV9 |
| Spinal muscular atrophy | Corticosteroids, eculizumab | NCT03306277 | Zolgensma | scAAV9 |
| Spinal muscular atrophy | Corticosteroids, eculizumab | NCT03461289 | Zolgensma | scAAV9 |
| Spinal muscular atrophy | Corticosteroids, eculizumab | NCT03505099 | Zolgensma | scAAV9 |
| Spinal muscular atrophy | Corticosteroids, eculizumab | NCT03837184 | Zolgensma | scAAV9 |
| Spinal muscular atrophy | Corticosteroids, eculizumab | NCT05073133 | AVXS-101 | scAAV9 |
| X-linked myotubular myopathy | Prednisolone | NCT03199469 | AT132 | AAV8 |
| X-linked retinoschisis | Corticosteroids | NCT02317887 | AAV8-scRS/IRBPhRS | AAV8 |
| X-linked retinitis pigmentosa | Corticosteroids | NCT03116113 | BIIB112 | AAV8 |