Trials
Haemophilia Gene Therapy Trials Database
Characteristics
Database last updated : 11 Oct 2024
Biological:
All
AAV2, FIX-WT, AAV-hF.IX, NCT00076557
AAV2, FIX-WT, rAAV-hAAT-F.IX
AAV5, FIX-Padua, Hemgenix, NCT03489291
AAV5, FIX-Padua, Hemgenix, NCT03569891 (Phase III, Hope-B Trial)
AAV5, FIX-WT, AMT-060, uniQure, NCT02396342
AAV5, FVIII-BDD, Roctavian, NCT02576795 (Phase 1/2 Study)
AAV5, FVIII-BDD, Roctavian, NCT03370913 (Phase 3, GENEr8-1 Study)
AAV5, FVIII-BDD, Roctavian, NCT03370913 and NCT03392974, Data of Participants with HIV
AAV6, FIX-WT, SB-FIX, NCT02695160 (No Results Published)
AAV6, FVIII-BDD, Giroctocogene Fitelparvovec (SB-525), NCT03061201 (Phase 1/2)
AAV6, FVIII-BDD, Giroctocogene Fitelparvovec (SB-525), NCT04370054 (Phase 3 study)
AAV8, FIX-Padua, BAX 335, NCT01687608
AAV8, FIX-WT, scAAV2/8-LP1-hFIXco, NCT00979238
AAV8, FVIII-BDD, SHP654 (BAX 888), NCT03370172
AAV8, FVIII-V3, AAV2/8-HLP-FVIII-V3, NCT03001830
AAVhu37, FVIII-BDD, DTX201, NCT03588299
AAVrh10, FIX-WT, DTX101, NCT02618915
AAVS3, FIX-Padua, FLT180a, NCT03369444
AAV-Spark100, FIX-Padua, Fidanacogene elaparvovec (ᴾʳBEQVEZ™), NCT02484092
AAV-Spark100, FIX-Padua, Fidanacogene elaparvovec (ᴾʳBEQVEZ™), NCT03861273 (Phase 3, BeneGene-2)
AAV-SPK200, FVIII-BDD, Dirloctocogene samoparvovec (SPK-8011), NCT03003533 and NCT03432520
Bioengineered AAV capsid, FVIII-BDD, SPK-8016, NCT03734588 and NCT03432520
LV, FVIII, YUVA-GT-F801, NCT03217032 (no results published)
LV, FVIII-ET3, CD68-ET3-LV CD34+, Expression Therapeutics, NCT04418414 (no results published)
Sponsor:
All
Avigen¹/CHOP ²
Baxalta now part of Shire
Bayer/Ultragenyx
BioMarin Pharmaceutical
CSL Behring
Dimension Therapeutics
Expression Therapeutics, LLC
Non-commercial
Non-commercial, Avigen/CHOP
Pfizer
Pfizer/Sangamo
Roche/Spark Therapeutics
Sangamo Therapeutics
Shenzhen Geno-Immune Medical Institute
Shire
Spark Therapeutics
uniQure
University College, London
Disorder:
All
Haemophilia A
Haemophilia B
Gene Therapy Trials
Biological
Sponsor
Disorder
AAV2, FIX-WT, AAV-hF.IX, NCT00076557
Avigen¹/CHOP ²
Haemophilia B
AAV2, FIX-WT, rAAV-hAAT-F.IX
Non-commercial, Avigen/CHOP
Haemophilia B
AAV5, FIX-Padua, Hemgenix, NCT03489291
uniQure
Haemophilia B
AAV5, FIX-Padua, Hemgenix, NCT03569891 (Phase III, Hope-B Trial)
CSL Behring
Haemophilia B
AAV5, FIX-WT, AMT-060, uniQure, NCT02396342
uniQure
Haemophilia B
AAV5, FVIII-BDD, Roctavian, NCT02576795 (Phase 1/2 Study)
BioMarin Pharmaceutical
Haemophilia A
AAV5, FVIII-BDD, Roctavian, NCT03370913 (Phase 3, GENEr8-1 Study)
BioMarin Pharmaceutical
Haemophilia A
AAV5, FVIII-BDD, Roctavian, NCT03370913 and NCT03392974, Data of Participants with HIV
BioMarin Pharmaceutical
Haemophilia A
AAV6, FIX-WT, SB-FIX, NCT02695160 (No Results Published)
Sangamo Therapeutics
Haemophilia B
AAV6, FVIII-BDD, Giroctocogene Fitelparvovec (SB-525), NCT03061201 (Phase 1/2)
Pfizer/Sangamo
Haemophilia A
AAV6, FVIII-BDD, Giroctocogene Fitelparvovec (SB-525), NCT04370054 (Phase 3 study)
Pfizer/Sangamo
Haemophilia A
AAV8, FIX-Padua, BAX 335, NCT01687608
Baxalta now part of Shire
Haemophilia B
AAV8, FIX-WT, scAAV2/8-LP1-hFIXco, NCT00979238
Non-commercial
Haemophilia B
AAV8, FVIII-BDD, SHP654 (BAX 888), NCT03370172
Shire
Haemophilia A
AAV8, FVIII-V3, AAV2/8-HLP-FVIII-V3, NCT03001830
University College, London
Haemophilia A
AAVhu37, FVIII-BDD, DTX201, NCT03588299
Bayer/Ultragenyx
Haemophilia A
AAVrh10, FIX-WT, DTX101, NCT02618915
Dimension Therapeutics
Haemophilia B
AAVS3, FIX-Padua, FLT180a, NCT03369444
University College, London
Haemophilia B
AAV-Spark100, FIX-Padua, Fidanacogene elaparvovec (ᴾʳBEQVEZ™), NCT02484092
Pfizer
Haemophilia B
AAV-Spark100, FIX-Padua, Fidanacogene elaparvovec (ᴾʳBEQVEZ™), NCT03861273 (Phase 3, BeneGene-2)
Pfizer
Haemophilia B
AAV-SPK200, FVIII-BDD, Dirloctocogene samoparvovec (SPK-8011), NCT03003533 and NCT03432520
Roche/Spark Therapeutics
Haemophilia A
Bioengineered AAV capsid, FVIII-BDD, SPK-8016, NCT03734588 and NCT03432520
Spark Therapeutics
Haemophilia A
LV, FVIII, YUVA-GT-F801, NCT03217032 (no results published)
Shenzhen Geno-Immune Medical Institute
Haemophilia A
LV, FVIII-ET3, CD68-ET3-LV CD34+, Expression Therapeutics, NCT04418414 (no results published)
Expression Therapeutics, LLC
Haemophilia A
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