TABLE 1.

Current FVIII AAV liver-directed gene therapy products for HA in clinical development.

Name	Sponsor	Vector serotype	Transgene‡	Manu-facturing	Phase	FVIII range (% normal)	ClinicalTrials.gov Identifier
Valoctocogene	Biomarin	AAV5	FVIII-SQ	Baculovirus/Insect cells	3	19–164	NCT03370913
roxaparvovec (BMN-270)							NCT03392974
SPK-8011	Spark	LK03	FVIII-SQ	Plasmid/Mammalian cells	1/2	<5–49	NCT03003533
SPK-8016	Spark	NA	FVIII-SQ	Plasmid/Mammalian cells	1/2	NA	NCT03734588
AAV2/8-HLP-FVIII-V3	UCL	AAV8	FVIII-V3	Plasmid/Mammalian cells	1/2	6–69	NCT03001830
SB-525	Sangamo	AAV6	FVIII-SQ	Baculovirus/Insect cells	1/2	4–150	NCT03061201
SHP654 (BAX888)	Shire	AAV8	FVIII-SQ	Plasmid/Mammalian cells	1/2	NA	NCT03370172
BAY 2599023 (DTX201)	Bayer	AAVhu37	FVIII-SQ	Plasmid/Mammalian cells	1/2	5–17	NCT03588299

^‡See (69) and reference therein for details about FVIII variants used in gene therapy. NA, not available; UCL, University College of London.