Taysha IPO Presentation Deck slide image

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 00 60
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