Table 5.
Current and Planned Gene Therapy Clinical Trials for Hemophilia A
| Sponsor | Treatment | Capsid Serotype | Promoter | Transgene Product | Dose (vg/kg) | Phase | Estimated Enrollment | Status | ClinicalTrials.gov Identifier |
|---|---|---|---|---|---|---|---|---|---|
| Bayer | BAY2599023 (DTX201) | hu37 | liver-specific | BDD-FVIII | n.d. | 1, 2 | 30 | recruiting | NCT03588299 |
| BioMarin | valoctocogene roxaparvovec (BMN 270) | AAV5 | HLP | BDD-FVIII | 6e12–6e13 | 1, 2 | 15 | active, not recruiting | NCT02576795 |
| 6e13 | 3 | 130 | recruiting | NCT03370913 | |||||
| 4e13 | 3 | 40 | recruiting | NCT03392974 | |||||
| valoctocogene roxaparvovec (BMN 270) in patients with AAV5 antibodies | 6e13 | 1, 2 | 10 | enrolling by invitation | NCT03520712 | ||||
| MCW | autologous CD34 + PBSC, modified with lentivirus encoding BDD-FVIII | – | – | – | – | 1 | 5 | not yet recruiting | NCT03818763 |
| Sangamo | SB-525 | AAV2/6 | liver-specific | BDD-FVIII | 9e11–3e13 | 1, 2 | 20 | recruiting | NCT03061201 |
| SGIMI | YUVA-GT-F801: autologous HSC/MSC modified with lentivirus encoding FVIII | – | – | – | – | 1 | 10 | not yet recruiting | NCT03217032 |
| Takeda | TAK-755 (formerly BAX 888/SHP654) | AAV8 | TTR | BDD-FVIII | n.d. | 1, 2 | 10 | recruiting | NCT03370172 |
| Spark | SPK-8011 | Spark200 | liver-specific | BDD-FVIII | 5e11–2e12 | 1, 2 | 30 | recruiting | NCT03003533 |
| SPK-8016 in patients with FVIII inhibitors | n.d. | BDD-FVIII | dose-finding | 1, 2 | 30 | recruiting | NCT03734588 | ||
| UCL/St. Jude | AAV2/8-HLP-FVIII-V3 | AAV2/8 | HLP | FVIII-V3 | 6e11–6e12 | 1 | 18 | recruiting | NCT03001830 |
BDD-FVIII, B-domain deleted clotting factor VIII; n.d., not disclosed; AAV, adeno-associated virus; HLP, hybrid liver-specific promoter; MCW, Medical College of Wisconsin; SGIMI, Shenzhen Geno-Immune Medial Institute; TTR, transthyretin; UCL, University College London; FIX, clotting factor IX; ApoE/hAAT, apolipoprotein E enhancer/human alpha 1-antitrypsin promoter; ZFN, zinc finger nuclease.