Taysha IPO Presentation Deck

TSHA-102 for the treatment of Rett syndrome to submit IND by end of 2021 Study design and duration Key inclusion/exclusion criteria Intervention Key clinical assessments Open-label, dose-ranging, multi-center Phase 1/2 trial Stage II and III Rett patients Age 3-10 (inclusive) Pathogenic confirmation of mutation in MECP2 • Low-dose cohort (n=4): single total dose of 5x1014 vg of AAV9/MECP2-miRARE High-dose cohort (n=4): single total dose of 1x1015 vg of AAV9/MECP2-miRARE Delivered intrathecally • Safety and tolerability Rett Syndrome Clinical Severity Score Rett Syndrome Behavior Questionnaire Seizure frequency/medications • Clinical Global Impression • Caregiver burden and Qol . Cardiac assessments (QT interval) • Head circumference & other growth parameters Respiratory assessments and sleep apnea, including in-patient sleep study T 37

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