Table 3.
In vivo gene therapy vectors under development for the treatment of LSDs.
| LSD | Drug Name and Trial ID | Action Mechanism | Trial Phase |
|---|---|---|---|
| FD | FLT190 (NCT04040049) | AAV vector (AAV8) which drive the GLA functional gene in the liver | I/II |
| 4D-310 (NCT04519749) | AAV based vector, which express GLA gene under CAG promoter action | I/II | |
| ST920 (NCT04046224) | vector based on AAV (AAV2/6) which drive the GLA functional gene in the liver | I/II | |
| PD | SPK-3006 (NCT04093349) | AAV based vector to express GAA in liver | I/II |
| Raav9-DES-Hgaa (NCT02240407) | Intramuscular AAV9, to express GAA | I/II | |
| AT845 (NCT04174105) | Intravenous AAV8 to express GAA | I/II | |
| MLD | TYF-ARSA (NCT03725670) | Self-inactivating lentiviral vector injected intracerebrally. The vector transports a correct version of ARSA gene. | I/II |
| MPSI | RGX-111 (NCT03580083) | Intracisternal, intracerebroventricular or lumbar puncture of AAV9 which express IDUA | I/II |
| SB913 (NCT03041324) (NCT04628871) | Permanent expression of iduronidase in hepatocytes, obtained by in vivo genetic editing in albumin locus driven by AAV | I/II | |
| MPSII | RGX-121 (NCT0457190) | AAV9-based vector which directs iduronate-2-sulfatase expression | I/II |
| SBFIX | Permanent expression of iduronate-2-sulphatase in hepatocytes, obtained by in vivo genetic editing from albumin locus directed by AAV-based vectors | I/II | |
|
MPSIII
(A, B) |
LYS-SAF302 | AAVrh10 intracerebral injection which express SGSH gene | I/II |
| SAF-301 (NCT02053064) | AAV10 intracerebral injection that express SGSH and SUMF1 genes | I/II | |
| ABO101 (NCT04655911) | AAV9 which express NAGLU gene by intracerebral injection | I/II | |
| MPS VI | AAV2/8.TBG.hARSB | AAV8 to express ARSB gene in liver | I/II |
| Krabbe Disease | AAVrh10 | AAV vector expressing galactosylceramidase is combined with HSCT and delivered intravenously | I/II |
| AAV Hu68 | Intracisternal AAV vector expressing galactosylceramidase | I/II |