TABLE 3.
Gene therapy clinical trials for MPS III
| Disease | Approach | Investigational medicinal product | Study status | Sponsor | Route of administration | Clinical trial identifier | Publications |
|---|---|---|---|---|---|---|---|
| MPS IIIA | AAV gene therapy | AAVrh.10‐SGSH‐IRES‐SUMF1 (SAF‐301) | Completed | Lysogene | Intraparenchymal | NCT01474343 | 103 |
| AAVrh.10‐h.SGSH (LYS‐SAF302) | Recruiting | Lysogene | Intraparenchymal | NCT03612869 | |||
| scAAV9.U1a.hSGSH (ABO‐102) | Recruiting | Abeona Therapeutics | Intravenous | 106 | |||
| AAVrh.9‐hSGSH (EGT‐101) | N/A | Esteve | ICV | N/A | 135 | ||
| Lentiviral gene therapy | Autologous CD34+ cells transduced with LV.CD11b.hSGSH (OTL‐201) | Recruiting |
University of Manchester Orchard Therapeutics |
Intravenous | NCT04201405 | 124 | |
| MPS IIIB | AAV gene therapy | AAV5‐hNAGLU | Completed | UniQure Biopharma | IP | NCT03300453 | 114 |
| AAV9.CMV.hNAGLU (ABO‐101) | Recruiting | Abeona Therapeutics | Intravenous | NCT03315182 | 117 |
Abbreviations: AAV adeno‐associated virus; ICV, intracerebroventricular; IP, intraparenchymal; LV, lentivirus; MPS III, mucopolysaccharidosis type III; SUMF1, sulfatase modifying factor 1.