Ultragenyx Invests 62% of Expenses in R&D, Advances Five Rare Disease Therapies
RARE•Ultragenyx advanced five investigational therapies in pivotal 2025 programs and invested roughly 62% of operating expenses in R&D to support trials at over 160 sites in 19 countries. The company expanded approved treatment access to 700+ patients in 50 nations and launched Act for Ultra-Rare advocacy.
1. Clinical Pipeline Progress
Ultragenyx advanced five investigational therapies through pivotal clinical programs in 2025, leveraging its Dynamic Development Model to engage patients and caregivers and executing trials at over 160 sites across 19 countries.
2. R&D Investment and Trial Reach
The company allocated approximately 62% of its operating expenses to research and development, driving accelerated progress and potentially reaching tens of thousands of patients with novel treatments for rare genetic diseases.
3. Patient Access and Advocacy
Expanded access initiatives supported more than 700 patients in 50 countries via patient assistance programs since 2013, and the new Act for Ultra-Rare policy campaign aims to foster legislation for ultra-rare disease treatments.
4. Sustainability and Governance
Corporate responsibility efforts included near-100% renewable electricity at key sites, waste reduction programs, a first Climate Risk Assessment, and board-level governance oversight, with no material data privacy breaches reported.
