Current rAAV FVIII gene transfer clinical trials
| Sponsor . | Intervention . | Gene Product . | Capsid . | Manufacturing platform . | Genome configuration . | Dose (vg/kg) . | Required steroids . | FVIII:C activity (% of normal) . | Identifier . | Status . | References . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Bio Marin Pharmaceuticals | BMRN-270 | BDD-FVIII | rAAV5 | Insect | ss | 6 × 1013 | 7/7 | 4-270 | NCT02576795 | Recruiting | 24 |
| Spark Therapeutics | SEK-8011 | BDD-FVIII | rAAV-Spark200 | Mammalian | ss | NCT03003533 | Recruiting | N/A | |||
| University College London | AAV2/8-HLP-FVIII-V3 | BDD-FVHIII | rAAV8 | Mammalian | ss | NCT03001830 | Not yet recruiting | N/A | |||
| Sangamo Biosciences | SB-525 | BDD-FVHIII | rAAV6 | Insect | ss | NCT03061201 | Not yet recruiting | N/A |
| Sponsor . | Intervention . | Gene Product . | Capsid . | Manufacturing platform . | Genome configuration . | Dose (vg/kg) . | Required steroids . | FVIII:C activity (% of normal) . | Identifier . | Status . | References . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Bio Marin Pharmaceuticals | BMRN-270 | BDD-FVIII | rAAV5 | Insect | ss | 6 × 1013 | 7/7 | 4-270 | NCT02576795 | Recruiting | 24 |
| Spark Therapeutics | SEK-8011 | BDD-FVIII | rAAV-Spark200 | Mammalian | ss | NCT03003533 | Recruiting | N/A | |||
| University College London | AAV2/8-HLP-FVIII-V3 | BDD-FVHIII | rAAV8 | Mammalian | ss | NCT03001830 | Not yet recruiting | N/A | |||
| Sangamo Biosciences | SB-525 | BDD-FVHIII | rAAV6 | Insect | ss | NCT03061201 | Not yet recruiting | N/A |
ss, Single stranded.