Ultragenyx Underscores Continued Commitment to Rare Disease Innovation with 2024 Impact Report

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Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc.

NOVATO, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2024 Impact Report today, which covers the company’s approach to corporate responsibility. The report highlights efforts to transform the lives of individuals and families affected by rare diseases, empower its employees, and make a meaningful impact on the communities it operates within and serves.

“Since founding Ultragenyx 15 years ago, I’ve sought to build a next-generation rare disease company that has a meaningful and sustained impact on the entire rare disease community,” said Emil Kakkis, M.D., Ph.D., founder, president, and chief executive officer of Ultragenyx. “Guided by the principle of doing the right thing, we have engineered our own dynamic approach to drug development while working on broader education and policy initiatives that meaningfully support diagnosis, drug development, regulatory pathways, and clinical care. The sustainability and social impact implications of our actions and decisions are consistently considered and reflected in our 2024 Impact Report.”

The company’s approach to corporate responsibility is structured around six key pillars—Innovation, Patients, People, Communities, Planet, and Governance—aligned with its vision to lead the future of rare disease medicine and its mission to transform the lives of people with rare diseases. The 2024 Ultragenyx Impact Report features updates for all six pillars, as well as Sustainability Accounting Standards Board (SASB) and Global Reporting Initiative (GRI) indices. For the full report and more on Ultragenyx’s Corporate Responsibility efforts, visit https://www.ultragenyx.com/ultra-committed/corporate-responsibility/.

Highlights from the company’s 2024 report include:

Innovation:
Ultragenyx is committed to developing and delivering transformative treatments where none exist.

  • Successfully transferred the manufacturing process of our DTX401 gene therapy to our Gene Therapy Manufacturing Facility (GTMF) in Bedford, Massachusetts

  • Advanced several key clinical programs to treat rare and ultra-rare diseases, including osteogenesis imperfecta (OI), Angelman syndrome, Sanfilippo syndrome type A (MPS IIIA), and glycogen storage disease type Ia (GSDIa)

  • Supported more than 80 clinical and non-clinical investigator-sponsored trials (ISTs) globally

Patients:
Ultragenyx is committed to supporting the rare disease community through our efforts to develop novel therapies, share our science and expertise, achieve broad access to screening and treatment, and partner with policymakers for meaningful change.