Current rAAV-mediated FIX gene transfer clinical trials
| Sponsor . | Intervention . | Gene product . | Capsid . | Manufacturing platform . | Genome configuration . | Highest-dose cohort (vg/kg) . | Required steroids . | Mean sustained FIX:C (% of normal) . | Identifier . | Status . | References . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| UCL/St. Jude’s | scAAV2/8-LP1-hFIXco | FIX | rAAV8 | Mammalian | sc | 2 × 1012 | 4/6 | 5.1 | NCT00979238 | Recruiting | 1,2 |
| Shire | AskBio | FIX-Padua | rAAV8 | Mammalian | sc | 3 × 1012 | 2/3 | 0 | NCT01687608 | Active, not recruiting | 20 |
| Spark Therapeutics/Pfizer | SPK-9001 | FIX-Padua | rAAV-Spark100 | Mammalian | ss | 5 × 1011 | 2/10 | 33 | NCT02484092 | Recruiting | 22 |
| uniQure | AMT-060 | FIX | rAAVS | Insect | sc | 2 × 1013 | 2/5 | 6.9 | NCT02396342 | Active, not recruiting | 23 |
| Dimension Therapeutics | DTX101 | FIX | rAAVrh-10 | Mammalian | sc | 5 × 1012 | 3/3 | 6.7 | NCT02971969 | Recruiting* | 36 |
| Sangamo Biosciences | SB-FIX | Zn finger–FIX | rAAV6 | Insect | NCT02695160 | Recruiting | N/A | ||||
| Freeline Therapeutics | FLT-180 | FIX-Padua | rAAV-engineersd | Mammalian | N/A |
| Sponsor . | Intervention . | Gene product . | Capsid . | Manufacturing platform . | Genome configuration . | Highest-dose cohort (vg/kg) . | Required steroids . | Mean sustained FIX:C (% of normal) . | Identifier . | Status . | References . |
|---|---|---|---|---|---|---|---|---|---|---|---|
| UCL/St. Jude’s | scAAV2/8-LP1-hFIXco | FIX | rAAV8 | Mammalian | sc | 2 × 1012 | 4/6 | 5.1 | NCT00979238 | Recruiting | 1,2 |
| Shire | AskBio | FIX-Padua | rAAV8 | Mammalian | sc | 3 × 1012 | 2/3 | 0 | NCT01687608 | Active, not recruiting | 20 |
| Spark Therapeutics/Pfizer | SPK-9001 | FIX-Padua | rAAV-Spark100 | Mammalian | ss | 5 × 1011 | 2/10 | 33 | NCT02484092 | Recruiting | 22 |
| uniQure | AMT-060 | FIX | rAAVS | Insect | sc | 2 × 1013 | 2/5 | 6.9 | NCT02396342 | Active, not recruiting | 23 |
| Dimension Therapeutics | DTX101 | FIX | rAAVrh-10 | Mammalian | sc | 5 × 1012 | 3/3 | 6.7 | NCT02971969 | Recruiting* | 36 |
| Sangamo Biosciences | SB-FIX | Zn finger–FIX | rAAV6 | Insect | NCT02695160 | Recruiting | N/A | ||||
| Freeline Therapeutics | FLT-180 | FIX-Padua | rAAV-engineersd | Mammalian | N/A |
N/A, not applicable; sc, self-complementary; ss, single stranded; UCL, University College London.
*Sponsor’s press release indicates the trial is no longer recruiting.