Table 1 Clinical trials of ex-vivo and in-vivo gene therapy (GT) in mucopolysaccharidosis (MPS)
From: Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches
Disease | Study phase | Type of vector | Route | No. of treated patients | Outcome | Sponsor |
|---|---|---|---|---|---|---|
In vivo | ||||||
 MPS IIIA | I/II | AAVrh.10-SGSH-IRES-SUMF1 | IC | 4 | Improvement in behaviour; brain atrophy stable/worse | Lysogene |
 MPS IIIA | II/III | AAVrh.10-SGSH | IC | In preparation | – | Lysogene |
 MPS IIIA | I/II | AAV9-SGSH | Intra-CSF | Starting enrolment | – | ESTEVE & UAB |
 MPS IIIA | I/II | AAV9-SGSH | Systemic | Starting enrolment | – | Abeona Therapeutics |
 MPS IIIB | I/II | AAV9-NAGLU | Intra-CSF | In preparation | – | ESTEVE & UAB |
 MPS IIIB | I/II | AAV9-NAGLU | Systemic | In preparation | – | Abeona Therapeutics |
 MPS I | I/II | AAV9-SGSH | Intra-CSF | In preparation | – | REGENXBIO |
 MPS I | I/II | AAV-ZFN | Systemic | Starting enrolment (for attenuated form) | – | Sangamo Therapeutics |
 MPS II | I/II | AAV9-SGSH | Intra-CSF | In preparation | – | ESTEVE & UAB; REGENXBIO |
 MPS II | I/II | AAV-ZFN | Systemic | Starting enrolment (for attenuated form) | – | Sangamo Therapeutics |
 MPS VI | I/II | AAV8-ARSB | Systemic | Starting enrolment | – | Fondazione Telethon |
Ex vivo | ||||||
 MPS IH | I/II | LV | IV | In preparation | – | Fondazione Telethon |
 MPS IIIA | I/II | LV | IV | In preparation | – | Orchard Therapeutics |