Investor Presentaiton

Gene therapy: AAV is by far the Most Popular Gene Delivery Vehicle; Rare Diseases and Retinal Diseases are Common Targeted Indications Gene therapy pipeline breakdown by cell type and phase 1200 BLA/ approval 1000 Ph3 Ph2/3 Ph1/2 Ph2 800 Ph1 IND 600 400 PCC Majority AAV-based 200 0 Conventional gene therapy Gene editing Others Source: PharmCube NextPharma database; Date cutoff 05/2023 Pipeline breakdown by indication 0% 2% 4% ALS DMD WAMD Hemophilia A Retinitis pigmentosa Parkinson's disease AMD Cystic fibrosis Alzheimer's disease Hemophilia B Leber's congenital... Friedreich's ataxia SMA A1AT-deficiency Pompe disease Stargardt's disease Diabetic macular edema 3 药明康德

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