Taysha IPO Presentation Deck
Angelman Syndrome is a rare neurodevelopmental
disorder with multiple genetic mechanisms
Angelman syndrome is caused by the loss of function
of the maternal copy of the UBE3A gene
Symptoms of AS include severe cognitive
dysfunction, absent speech, seizures, ataxia, gait
disorder, microcephaly, and happy affect
The paternal copy of UBE3A is normal, but silenced,
in all cases of AS
Taysha is developing gene therapies to activate the
paternal UBE3A using an shRNA construct
The estimated prevalence of Angelman syndrome is
30,000 patients in the US and EU
AAV9+
IXAT
Anti-UBE3A-
ATS shRNA
Intrathecal
delivery
Maternal Paternal
Maternal Paternal
Ube3a-ATS
noroor
MMMM Ube 3a
NMMMM Uka MIX
TAYSHA
Ube
MMMM Ube3a MX
NMNMNMNMUDca
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