Work packages
| WP Number | WP Title | Lead beneficiary | |
|---|---|---|---|
| 1 | Production of clinical-grade AAV2/8.TGB.hARSB | ReGenX | |
| 2 | Pre-clinical evaluation of the AAV2/8.TBG.hARSB GMP vector | FTELE.IGM | |
| 3 |
Regulatory filing |
FTELE.IGM | |
| 4 |
Multicentre Phase I/II MPS VI clinical trial |
DISMET | |
| 5 |
Ethics and Patient Organizations |
DISMET | |
| 6 |
Management |
FTELE.IGM | |
| 7 |
Dissemination |
FTELE.IGM | |
| 8 |
Scientific coordination |
FTELE.IGM |
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