Taysha IPO Presentation Deck
Novel bicistronic vector design allows consistent
expression of HEXA and HEXB genes
HEXA and HEXB genes are required to produce
the subunits of the beta-hexosaminidase A
enzyme
The novel bicistronic vector design enables 1:1
expression of HEXA and HEXB transgenes under
the control of a single promoter with a P2A-self-
cleaving linker
- SD mice received vehicle or varying doses of
TSHA-101 after 6 weeks:
High dose (2.5x10¹1¹ vg/mouse)
Medium dose (1.25x10¹¹ vg/mouse)
Low dose (0.625x10¹1 vg/mouse)
- Vehicle controls
SSAAV ITR
F.
CAG
Drive high levels of
gene expression
Full copies of hHEXB and hHEXA genes
separated by P2A self-cleaving linker
hHEXB
P2A
AAV9 capsid
Brain tropism &
favorable safety profile
*
hHEXA
SSAAV ITR
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