Table 2.
Hemophilia A gene therapy trial using AAV
| Sponsor | Transgene | Vector | Outcomes | Current status |
|---|---|---|---|---|
| BioMarin Pharmaceutical (BMN 270) | Codon-optimized BDD-FVIII | AAV5 | 6e13 vg/kg: FVIII expression in the normal range for 6 of 7 patients. | Closed |
| 4e13 vg/kg: FVIII expression in the mild range for 6 of 6 patients. | ||||
| Transient transaminitis at 6-20 wk after gene transfer in 8 of 9 patients | ||||
| UCL/St. Jude (GO-8) | Codon-optimized FVIII; B domain replaced with V3 peptide | AAV8 | Completed recruitment to low (6e11 vg/kg) and intermediate (2e12 vg/kg) dose levels. FVIII expression = 8-64%. | Open |
| Transient transaminitis in 2 of 3 patients enrolled | ||||
| Spark Therapeutics (SPK-8011) | BDD-FVIII | AAV-LK03 | Completed recruitment to low-dose (5e11 vg/kg), intermediate-dose (1e12 vg/kg), and high-dose (2e12 vg/kg) levels. FVIII expression = 7-30%. | Open |
| Sangamo Bioscience (SB-525) | BDD-FVIII | AAV6 | Enrolled 2 patients in each cohort (9e11, 2e12, 1e13, and 3e13 vg/kg). FVIII expression in the highest-dose cohort = 94-140%. | Open |
| Ultragenyx Therapeutics (DTX-201 | BDD-FVIII | AAVhu37 | Trial open | |
| Takeda (TAK 754) | BDD-FVIII | AAV8 | Trial open | |