Table 1.
Ongoing hemophilia A and B gene therapy trials
| Sponsor | Clinicaltrials.gov | Manufacturing platform | Transgene | Capsid serotype | Dose (vg/kg) | Phase | Ref |
|---|---|---|---|---|---|---|---|
| HB | |||||||
| UCL/St. Jude | NCT00979238 | Mammalian | scFIX | AAV8 | 2 × 1011 to 2 × 1012 | 1/2 | 16,45 |
| Pfizer/Spark | NCT03681273 NCT03307980 |
Mammalian | ssFIX-R338L | SPK100 | 5 × 1011 | 1/2, 3 | 9 |
| uniQure | NCT02396342 | Sl9 (insect) | ssFIX-R338L | AAV5 | 2 × 1013 | 3 | 8 |
| Freeline | NCT03369444 | Mammalian | scFIX-R338L | AAVS3 | 7.5 × 1011 9.5 × 1011 | 10 | |
| HA | |||||||
| BioMarin |
NCT02576795 NCT03392974 NCT03370913 |
Sl9 (insect) | ssFVIII-SQ | AAV5 | 6 × 1013 | 1/2, 3 | 11,15 |
| Sangamo/Pfizer | NCT03061201 | Sl9 (insect) | ssFVIII-SQ | AAV6 | 3 × 1013 | 1/2, 3 | 18 |
| Spark |
NCT03003533 NCT03432520 |
Mammalian | ssFVIII-SQ | LK03 | 5 × 1011 to 2 × 1012 | 1/2 | 14 |
| UCL/St. Jude | NCT03001830 | Mammalian | ssFVIII-V3 | AAV8 | 6 × 1011 to 6 × 1012 | 1/2 | 4,5 |
| Bayer/Ultragenyx | NCT03588299 | Mammalian | ssFVIII-SQ | AAVhu37 | 5 × 1012 to 2 × 1013 | 1/2 | 58 |
| Takeda | NCT03370172 | Mammalian | ssFVIII-SQ | AAV8 | 1/2 | ||
sc, self-complementary; ss, single stranded; UCL, University College London.