Ultragenyx Analyst Day Presentation

Four pivotal gene therapy programs DTX401 for GSDla • HEK293 manufacturing process . Phase 3 fully enrolled • Data anticipated in 1H24 . DTX301 for OTC HEK293 manufacturing process ⚫ Phase 3 FPI in February 2023 Expect Ph3 to be fully enrolled in 1H24 15 Confidential and Proprietary M UX111 for MPS IIIA • HEK293 manufacturing process • Updated pivotal data presented at WORLDSymposium TM in February 2024 Seeking accelerated review path with FDA UX701 for Wilson disease • Pinnacle PCLTM manufacturing process Phase 1/2/3 study ongoing Stage 1 enrollment completed in January 2024 & data expected mid-2024 ultragenyx

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