Ultragenyx Analyst Day Presentation

Key licenses & intellectual property - Clinical programs. Product License UX143 Mereo Biopharma (Osteogenesis Imperfecta) DTX401 (GSDIa) US Intellectual Property Rights/Royalties + Milestones Setrusumab antibody (2028) ° Use of anti-sclerostin antibodies including setrusumab for treatment of OI (2037) ° • Tiered double-digit royalty on ex-EU sales and clinical, regulatory, and commercial milestones to Mereo Fixed double-digit royalty on EU sales to Ultragenyx ° Sub-License from REGENXBIO of UPENN IP AAV8 Capsid (2024) • NIH (Non-Exclusive) • Low to mid single-digit royalty and development milestones Recombinant vectors comprising codon-optimized G6Pase gene (2034) Low single-digit royalty Recombinant vectors comprising SGSH gene (Pending; 2032) UX111/ABO-102 Nationwide Children's Hospital (NCH) • (MPS IIIA) Development milestones up to $1M plus low single-digit royalty Abeona Therapeutics DTX301 Sub-License from REGENXBIO of UPENN IP ° (OTC Deficiency) Sub-License from REGENXBIO of UPENN IP • UX701 (Wilson Disease) UPENN N/A (IP Owned by Ultragenyx) Texas A&M University GTX-102 (Angelman Syndrome) GeneTx 21 Confidential and Proprietary • • Commercial milestones up to $30M plus tiered royalty up to 10% AAV8 Capsid (2024) Recombinant vectors comprising codon-optimized OTC gene (2035) Low to mid single-digit royalty and development milestones AAV9 Capsid (2024-2026) Mid to high single-digit royalty and up to $9M in development milestones Recombinant vectors comprising certain regulatory and coding sequences packaged in UX701 (2039) Development up to $5M and commercial milestones up to $25M plus low to mid single-digit royalty Recombinant vectors expressing a novel truncated version of ATP7B protein produced by UX701 (Pending; 2040) Use of UBE3A-ATS antisense oligonucleotides including GTX-102 for treatment of AS (2038) Development and commercial milestones plus mid single-digit royalty Development, regulatory, and commercial milestones up to $190M plus mid to high single-digit royalty ultragenyx

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