Taysha IPO Presentation Deck
Development of a gene therapy for Rett syndrome
requires regulated expression of MECP2
AAV9/MECP2 caused dose-dependent side effects
after intraCSF administration in WT and KO mice
We have developed a novel miRNA-responsive
target sequence (miRARE) that regulates the
expression of the MECP2 transgene
Our approach provides a superior therapeutic
profile to that of competitor unregulated MECP2
gene replacement
Enables rapid onset
and stable expression
SCAAV ITR MP426
miniMECP2 gene with miRARE panel to regulate
MECP2 expression
miniMECP2
Fragment of the endogenous MECP2 promoter
that mimics cell-specificity of MECP2
AAV9 capsid
Brain tropism &
favorable safety profile
miRARE
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SCAAV ITR
Enables rapid onset
and stable expression
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