Taysha IPO Presentation Deck

Prader-Willi syndrome is caused by genetic errors on chromosome 15 Symptoms of PWS include neonatal hypotonia followed by hyperphagia and excessive weight gain that begins around 5-8 years of age and lasts until death Hyperphagia leads to severe obesity, metabolic disorder, and is a leading cause of morbidity and mortality Intellectual disability and developmental delay and psychiatric disorders are also common The average age of death is approximately 30 years The estimated prevalence of PWS is 40,000 patients in the US and EU AAV9 IXAT PWS shRNA Intrathecal delivery ubject ans of S HELLKLUM VERRER Number Selected clinical features reported for patients with Prader-Willi syndrome 3 WT Mat Feeding Difficulties SAUNA d usprucbredy Behavioral problems اسال SANGA/REMA ATS typopigmentation Eye Abnormalites Deliver targeted shRNA utilizing AAV9 to activate maternal gene expression Speech Defects 1999 Present Absent No deta 62

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