Table 1.

Ongoing hemophilia A and hemophilia B AAV gene therapy trials

Sponsor	ClinicalTrials identifier	Manufactoring platform	Transgene	AAV Serotype	Dose (× 1011 vg/kg)	AAV antibody criteria	Phase	Reference
Hemophilia B
Pfizer/Spark	NCT02484092 NCT03307980	HEK293	ssFIX-R338L	SPK100	5	≤1:5	I/II, III	27
CSL/uniQure	NCT02396342 NCT03489291 NCT03569891	Sl9	ssFIX-R338L	AAV5	200	None	III	101
Freeline	NCT03369444	HEK293	scFIX-R338L	AAVS3	7.5–9.5	Negative	I/II	59
Hemophilia A
BioMarin	NCT02576795 NCT03392974 NCT03370913	Sl9	ssFVIII-SQ	AAV5	600	Negative	I/II, III	57, 61
Pfizer/Sangamo	NCT03061201	Sl9	ssFVIII-SQ	AAV6	300	Negative	I/II, III	60
Spark	NCT03003533 NCT03432520	HEK293	ssFVIII-SQ	LK03	5–20	≤1:5	I/II	56
UCL/St.Jude	NCT03001830	HEK293	ssFVIII-V3	AAV8	6–60	Negative	I/II	21
Bayer/Ultragenyx	NCT03588299	HEK293	ssFVIII-SQ	AAVhu37	50–200	Negative	I/II	102
Takeda	NCT03370172	HEK293	ssFVIII-SQ	AAV8	20–120	<1:5	I/II	102

Abbreviations: AAV, adeno-associated viral; UCL, University College London; sc, self-complementary; ss, single stranded; vg, vector genomes.