| Hemophilia B gene therapy pipeline |
| Company |
Project |
Mechanism |
Notes |
Trial details |
| Baxalta (Shire) |
BAX 335 |
AAV8-Padua FIX |
Phase III to start 2016 |
NCT01687608 |
| UniQure/Chiesi |
AMT-060 |
AAV5-wild-type FIX |
Low-dose phase I/II data presented at EHA; high-dose data H2 2016 |
NCT02396342 |
| Spark/Pfizer |
SPK-9001 |
Bio-engineered AAV-Padua FIX |
Low-dose phase I/II data presented at EHA |
NCT02484092 |
| Dimension Therapeutics/ Regenxbio |
DTX101 |
AAVrh10-wild-type FIX |
Initial phase I/II data due H2 2016 |
NCT02618915 |
| Sangamo Biosciences |
SB-FIX |
In vivo protein replacement using zinc finger nucleases |
Phase I trial started in 2016 |
NCT02695160 |
| Freeline Therapeutics |
hemophilia B Gene |
