NOVATO, Calif., July 08, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) released its 2025 Impact Report, detailing the company's work on behalf of patients and families living with rare and ultra-rare diseases. In 2025, Ultragenyx advanced five investigational therapies in pivotal clinical programs with the potential to reach tens of thousands of patients around the world - while continuing to build on years of work to support patients who cannot yet access approved treatments, which have now reached individuals in 50 countries.
"The rare disease community has waited long enough - every program we advance, every trial site we open, and every patient we support through access to approved medicines is a reflection of that belief," said Emil Kakkis, M.D., Ph.D., founder, president, and chief executive officer of Ultragenyx. "Our 2025 Impact Report reflects the progress we are making today to deliver urgently needed, first-ever therapies, as well as our commitment to shaping a more accessible future for rare diseases."
The report outlines progress across six key pillars - Innovation, Patients, People, Communities, Planet, and Governance - as well as Sustainability Accounting Standards Board (SASB), Global Reporting Initiative (GRI), and Task Force on Climate Related Disclosures (TCFD) indices. Updates reflect Ultragenyx's ongoing focus on delivering meaningful, long-term impact for patients and families impacted by rare and ultra-rare diseases and cover activities from January 1 through December 31, 2025. For the full report and more on Ultragenyx's Corporate Responsibility efforts, visit https://www.ultragenyx.com/ultra-committed/corporate-responsibility/.
Highlights from 2025 include:
Innovation
Ultragenyx continued to expand access to its four approved therapies across five indications and advanced its pipeline with a singular focus on addressing significant unmet medical needs for rare disease patients:
- Invested approximately 62% of operating expenses in research and development in 2025
- Drove progress on five clinical-stage investigational therapies with the potential to reach tens of thousands of patients
- Applied its Dynamic Development Model (DDM) - centered around direct patient and caregiver input - to inform clinical trial design and accelerate clinical development taking place across 160+ clinical trial sites in 19 countries
Patients
Ultragenyx remained committed to improving access, advancing advocacy, and partnering with the rare disease community.
- Hosted two Rare Bootcamps, including the first held on the U.S. East Coast, to support patient-led drug development initiatives
- Supported more than 700 patients in 50 countries through expanded access and patient assistance programs since 2013
- Participated in over 45 global patient advocacy events and engagements in 2025
- Launched Act for Ultra-Rare, initiating policy discussions to advocate for legislation that supports the development of treatments for ultra-rare diseases to help ensure that these patient communities are not left behind
People
Ultragenyx invested in its global workforce through engagement, inclusion, and professional development.
- Maintained strong employee engagement, with an overall engagement score of 86%
- Delivered more than 80 employee learning and development workshops in 2025
- Achieved 92% participation in the annual employee engagement survey
Communities
Ultragenyx expanded its philanthropic impact and community engagement efforts globally.
- Supported ~180 organizations across ~230 grant programs focused on education, awareness, and patient advocacy
- Contributed more than 2,800 employee volunteer hours supporting local and global initiatives and hosted the third annual Global Days of Service, encompassing nearly 40 volunteer projects worldwide
- Approved approximately $3.3 million in charitable donations, medical education, and health-related grants across more than 20 countries
Planet
Ultragenyx advanced environmental sustainability initiatives across its operations.
- Purchased ~100% renewable electricity for its Novato, CA headquarters campus and Bedford, MA Gene Therapy Manufacturing Facility, and diverted tens of thousands of pounds of waste through recycling and sustainable lab practices
- Initiated its second My Green Lab certification in Somerville, MA, building on a 'Green' level certification in Novato, CA
- Conducted its inaugural Climate Risk Assessment to evaluate physical and transition risks across operations and supply chain
Governance
Ultragenyx focused on strong corporate governance, compliance, and ethical business practices.
- Continued to integrate corporate responsibility oversight through board-level governance structures
- Maintained a comprehensive compliance program aligned with global regulatory standards
- Reported no material data privacy breaches or cybersecurity incidents in 2025
Recognition
Ultragenyx's achievements in 2025 were recognized through multiple awards and honors.
- Named a Top Place to Work in the USA and recognized by The Boston Globe and San Francisco Chronicle
- Received multiple Top Workplaces Culture Excellence Awards, including recognition for compensation, well-being, innovation, leadership, and professional development
- Honored for excellence in patient engagement, including recognition from the rare disease community
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
This press release and the 2025 Ultragenyx Impact Report and other materials cross-referenced in this press release contain statements that are aspirational or reflective of our views about the company's future performance that constitute "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are generally identified through the inclusion of words such as "aim," "anticipate," "aspire," "believe," commit," "endeavor," "estimate," "expect," "goal," "intend," "may," "plan," "seek," "strive," "target," "will," vision," "mission," "strategy," "commitment" and "work," or similar statements or variations of such terms and other similar expressions that predict or indicate future events or trends or that are not statements of historical fact. The forward-looking statements in this document and the materials cross-referenced concern Ultragenyx's goals, progress or expectations with respect to corporate responsibility, sustainability, patients, products, product candidates, employees, environmental matters, policy and business risks and opportunities and are not intended to create legal rights or obligations. Forward-looking statements inherently involve risks and uncertainties that could cause actual results to differ materially from those predicted in such statements including changes in economic conditions, slowed or insufficient technological developments, stakeholder engagement, changes in corporate strategy, and changes in the legal or regulatory environment. These statements are based on numerous assumptions that the company believes are reasonable but are open to a wide range of uncertainties and business risks. In addition, these statements may be based on standards for measuring progress that are still developing, controls and processes that continue to evolve, and assumptions that are subject to change in the future, and certifications, representations or data reviewed or provided by third parties Consequently, actual results may vary materially from what is contained in a forward-looking statement.
For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 6, 2026, and its subsequent periodic reports filed with the SEC. Forward-looking statements are aspirational and are not guarantees or promises that goals or targets will be met. Ultragenyx undertakes no obligation to update any forward-looking or other statements, whether as a result of new information, future events, or otherwise, and notwithstanding any historical practice of doing so. Ultragenyx may determine to adjust any goals and targets or establish new ones to reflect changes in its business. The information included in, and any issues identified as material for purposes of, the 2025 Ultragenyx Impact Report is not an indication that they are considered material to Ultragenyx, its investors or other stakeholders, or required to be disclosed in the company's filings, in each case under SEC reporting or any other laws or requirements that may apply to the company. In the context of this report, the term "material" is distinct from, and should not be confused with, such term as defined for SEC or other mandatory reporting purposes. Historical clinical trial success rates are not necessarily predictive, and should not be considered a guarantee, of future success rates.
In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx's Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).
Ultragenyx Contacts
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