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General Adeno-associated Viral Vector (AAV) Platforms Customized recombinant AAV (rAAV) genome construction and validation General AAV packaging, purification and tittering Premade AAV control vectors In vitro and in vivo transduction Fig 1. Customized recombinant AAV (rAAV) genome construction Fig 2. In vitro validation by two clinically-relevant rAAV vectors AAV-mediated Gene Replacement Therapy Customized therapeutic rAAV vector design, construction and packaging In vitro validation of the therapeutic effect Dose-escalation long-term efficacy and safety evaluation in vivo including wild type and disease models Fig 3. Schematic diagram of AAV-mediated Gene Replacement Therapy Fig4. Efficacy and safety evaluation of AAV8-Gene A in vivo. a. Biodistribution of AAV8-GFP in mice. b. Transduction efficiency of AAV8-GFP vector in liver. c. Gene A expression levels in mice at week 1, 2, 4, 6 and 8 post viral delivery. d. No alanine transaminase (ALT) change demonstrate the safety of the gene delivery. AAV-mediated In Vivo Genome Editing and Therapy Customized rAAV vector design , construction and packaging for in vivo genome editing In vitro validation of genome editing In vivo genome editing efficiency and functional evaluation Fig 5. Schematic diagram of in vivo genome editing and therapy Fig 6. Validation of in vivo KO by AAV8-sgGene B. a, Editing efficiency of AAV8-sgGene B. b, Representative indels formation in liver tissue. Business Contact: North America: Declan Ryan, declan.ryan@wuxiapptec.com Europe and Israel: Dave Madge, dave_madge@wuxiapptec.com China: Marcher Xu, xu_longji@wuxiapptec.com Japan: Yan Zhu, yan_zhu@wuxiapptec.com Korea: Suk Young Cho, sycho@wuxiapptec.com