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. Author manuscript; available in PMC: 2020 Jun 1.
Published in final edited form as: Gastroenterol Clin North Am. 2019 Apr 1;48(2):319–330. doi: 10.1016/j.gtc.2019.02.005

Table 1:

First generation of recombinant AAV serotype vectors used/being used for the potential gene therapy of hemophilia.

Investigators/
Sponsors		 Vector Dose Expression
level		 Total dose* Ref.
Hemophilia B High/Kay ssAAV2 8×1010 vgs/kg
4×1011 vgs/kg
2×1012 vgs/kg		 0%
0%
11%➔0%		 5.6 Trillion
28 Trillion
140 Trillion		 [36]
Nathwani/
Davidoff		 scAAV8 2×1011 vgs/kg
6×1011 vgs/kg
2×1012 vgs/kg		 2%
2-4%
8-12%➔5%		 14 Trillion
42 Trillion
140 Trillion		 [24,25]
Baxalta** scAAV8 2×1011 vgs/kg
1×1012 vgs/kg
3×1012 vgs/kg		 2-5%
20-25%
50%➔?%		 14 Trillion
70 Trillion
210 Trillion		 [65,69]
Spark
Therapeutics		 Modified
ssAAV8		 5×1011 vgs/kg 28-41 %➔26-33% 35 Trillion [25]
Dimension
Therapeutics*** 		ssAAVrh10 1.6×1012 vgs/kg
3×1012 vgs/kg		 <2%
<2%		 112 Trillion
210 Trillion		 [66 ]
uniQure scAAV5 5×1012 vgs/kg
2×1013 vgs/kg		 3-6.8%
3-12.7%		 350 Trillion
1.4 Quadrillion		 [26]
Hemophilia A BioMarin ssAAV5 2×1013 vgs/kg
6×1013 vgs/kg		 2-5%
50-200%➔32-59%		 1.4 Quadrillion
4.2 Quadrillion		 [27,67]
Spark
Therapeutics		 ssLK-03 5×1011 vgs/kg
1×1012 vgs/kg
2×1012 vgs/kg		 6-37%
7-24%
16-49%**** 		35 Trillion
70 Trillion
140 Trillion		 [68]
*

Based on an average patient’s weight of 70 kg (Estimated number of cells in a 70 kg “reference man” = 3 x 1013, or 30 Trillion) Data from Sender R, Fuchs S, Milo R. Revised estimates for the number of human and bacteria cells in the body. PLoS Biol 2016;14:e1002533 with permission.

**

Acquired by Shire.

***

Acquired by Ultragenyx.

****

Serious adverse events in 2 patients.