Gene Therapy Database
Characteristics
Database last updated : 11 Sep 2023
Gene Therapy Trials
Biological Sponsor Disorder
AAV2, FIX-WT, rAAV-hAAT-F.IX ³ Non-commercial, Avigen/CHOP ²⁸ Haemophilia B
AAV2, FIX-WT, AAV-hF.IX, NCT00076557 ²⁹ Avigen/CHOP ²⁸ Haemophilia B
AAV5, FIX-Padua, Hemgenix, NCT03569891 ³⁰ˑ ³¹ˑ ⁶² CSL Behring Haemophilia B
AAV5, FIX-Padua, Hemgenix, NCT03489291 ¹⁴ˑ ¹⁵ uniQure Haemophilia B
AAV5, FIX-WT, AMT-060, uniQure, NCT02396342 ¹¹⁻¹³ˑ ⁵⁸ uniQure Haemophilia B
AAV5, FVIII-BDD, Roctavian, NCT02576795 (Phase 1/2 Study) ¹⁸ˑ ¹⁹ˑ ³⁸ˑ ⁵⁹ BioMarin Pharmaceutical Haemophilia A
AAV5, FVIII-BDD, Roctavian, NCT03370913 (Phase 3 Study)⁵²ˑ ⁵³ 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 ²²⁻²³ˑ ³⁶ Pfizer/Sangamo Haemophilia A
AAV8, FIX-Padua, BAX 335, NCT01687608 ᵃ *ˑ ⁴⁻⁵ˑ ³⁴ˑ ³⁹ Baxalta now part of Shire *ˑ ²⁸ Haemophilia B
AAV8, FIX-WT, scAAV2/8-LP1-hFIXco, NCT00979238 ⁶ˑ ⁷ˑ ⁸ Non-commercial Haemophilia B
AAV8, FVIII-BDD, SHP654 (BAX 888), NCT03370172 Shire Haemophilia A
AAV8, FVIII-V3, AAV2/8-HLP-FVIII-V3, NCT03001830 * ²¹ University College, London ⁴³ Haemophilia A
AAVhu37, FVIII-BDD, DTX201, NCT03588299 *ˑ ²⁴ˑ ²⁵ˑ ⁵⁷ Bayer/Ultragenyx Haemophilia A
AAVrh10, FIX-WT, DTX101, NCT02618915 ¹⁻² Dimension Therapeutics Haemophilia B
AAVS3, FIX-Padua, FLT180a, NCT03369444 [16, 17] Freeline Therapeutics Haemophilia B
AAV-Spark100, FIX-Padua, Fidanacogene elaparvovec, NCT02484092 *ˑ ⁹ˑ ¹⁰ Pfizer * Haemophilia B
AAV-SPK200, FVIII-BDD, dirloctocogene samoparvovec (SPK-8011), NCT03003533 and NCT03432520 [1] Roche/Spark Therapeutics Haemophilia A
Bioengineered AAV capsid, FVIII-BDD [1], SPK-8016 [1], NCT03734588 [2] and NCT03432520 [3] 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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