Table 1. Summary of hemophilia gene... | American Society of Hematology

Table 1.

Summary of hemophilia gene transfer trials.

Sponsor	Trial No.	n	Vector/Route	Factor Level^†	Side Effects
Abbreviations: AAV2, adeno-associated virus, type 2; IM, intramuscular; IV, intravenous.
†Range of factor levels indicated; all patients were at < 1% before study.
^‡Transient elevated transaminase levels/dose-dependent.
Chiron	1—Phase I	13	Retrovirus/IV	0–1%	None
TKT	2—Phase I	6	Plasmid/omentum	0–4%	None
Avigen	3—Phase I	9	AAV2/IM	0–1%	None
Avigen	4—Phase I	6	AAV2/intrahepatic	3–12%	Elevated transaminase^‡
Genstar	5—Phase I	3	Adenovirus/IV	0–1%	Elevated transaminase,^‡ thrombocytopenia

Sponsor	Trial No.	n	Vector/Route	Factor Level^†	Side Effects
Abbreviations: AAV2, adeno-associated virus, type 2; IM, intramuscular; IV, intravenous.
†Range of factor levels indicated; all patients were at < 1% before study.
^‡Transient elevated transaminase levels/dose-dependent.
Chiron	1—Phase I	13	Retrovirus/IV	0–1%	None
TKT	2—Phase I	6	Plasmid/omentum	0–4%	None
Avigen	3—Phase I	9	AAV2/IM	0–1%	None
Avigen	4—Phase I	6	AAV2/intrahepatic	3–12%	Elevated transaminase^‡
Genstar	5—Phase I	3	Adenovirus/IV	0–1%	Elevated transaminase,^‡ thrombocytopenia