Mission Statement, Vision, & Core Values of Ultragenyx Pharmaceutical Inc. (RARE)

In the complex world of biopharmaceuticals, where innovation meets patient care, how does a company stay true to its purpose? With over 7,000 rare diseases affecting millions worldwide, companies like Ultragenyx stand at the forefront, striving to make a difference. But what exactly drives their commitment to developing treatments for these often-overlooked conditions?

Ultragenyx Pharmaceutical Inc. reported a $569 million net loss for 2024, yet their total revenue reached $560 million, marking a 29% increase from the previous year! How does a company balance financial realities with a mission to transform lives? Let's explore the core values, vision, and mission that guide Ultragenyx in its quest to bring hope to the rare disease community.

Ultragenyx Pharmaceutical Inc. (RARE) An Overview of

Founded in 2010, Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company committed to developing and commercializing novel products for the treatment of rare and ultra-rare genetic diseases. The company focuses on addressing unmet medical needs in serious genetic diseases where there are limited or no approved therapies. Their portfolio includes approved treatments and product candidates in various stages of clinical development, targeting metabolic, skeletal, and other genetic disorders.

As of April 2025, Ultragenyx continues to market several key products. While specific, real-time sales figures for April 2025 are not available, information from the company's latest financial reports provides insight into their financial performance.

In the latest reporting period, Ultragenyx has demonstrated strong financial performance, driven by key product sales and market growth. For example, in 2024, Ultragenyx reported record-breaking total revenue of $482.8 million, a 23% increase compared to the $393.2 million reported in 2023. This growth was primarily fueled by the strong performance of its leading products:

• Crysvita: Product revenue increased to $314.9 million in 2024 from $246.5 million in 2023, representing a 28% increase.
• Dojolvi: Product revenue increased to $77.8 million in 2024 from $59.2 million in 2023, representing a 31% increase.
• Mepsevii: Product revenue increased to $47.5 million in 2024 from $42.5 million in 2023, representing a 12% increase.

These figures highlight Ultragenyx's robust growth trajectory and market penetration in the rare disease treatment space. The company's strategic focus on developing and commercializing treatments for rare genetic disorders has positioned it as a key player in the biopharmaceutical industry.

Ultragenyx Pharmaceutical Inc. has emerged as a leader in the biopharmaceutical industry, dedicated to developing treatments for rare and ultra-rare genetic diseases. To understand the factors driving the company's success and its strategic direction, explore more here: Exploring Ultragenyx Pharmaceutical Inc. (RARE) Investor Profile: Who’s Buying and Why?

Ultragenyx Pharmaceutical Inc. (RARE) Mission Statement

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases. The company strives to address diseases with high unmet medical needs where there are typically no approved therapies that treat the underlying disease. Exploring Ultragenyx Pharmaceutical Inc. (RARE) Investor Profile: Who’s Buying and Why?

The company's mission to develop products for rare diseases is both noble and necessary. Ultragenyx's dedication to this cause has led to the development of groundbreaking therapies that have the potential to change the lives of patients with rare diseases.

Guided by the vision of a world where every patient has access to life-changing treatments, Ultragenyx is driven by core values of integrity, collaboration, and a relentless pursuit of excellence.

Recent highlights from Ultragenyx include:

• Total Revenue (2024): $560 million, a 29% increase compared to 2023.
• Crysvita Revenue (2024): $410 million, reflecting a 25% growth over 2023.
• Dojolvi Revenue (2024): $88 million, a 25% increase from the previous year.
• Evkeeza Revenue (2024): $32 million, showing strong growth following launches in regions outside the United States.

These figures demonstrate Ultragenyx's strong financial performance and commitment to expanding access to its therapies.

The company's financial goals for 2025 include:

• Total revenue between $640 million and $670 million, representing a 14% to 20% increase from 2024.
• Crysvita revenue between $460 million and $480 million, a 12% to 17% rise compared to 2024.
• Dojolvi revenue between $90 million and $100 million, indicating a 2% to 14% growth over the previous year.

These projections reflect the company's confidence in its continued growth and the increasing demand for its products.

Ultragenyx's corporate responsibility strategy is structured around six key pillars:

• Innovation
• Patients
• People
• Communities
• Planet
• Governance

These pillars guide the company's efforts to manage and communicate on topics relevant to its business and stakeholders.

Ultragenyx is dedicated to:

• Delivering novel, disease-modifying treatments with speed and urgency to rare disease communities with limited or no treatment options.
• Supporting the rare disease community through the development of novel therapies, sharing science and expertise, achieving broad access to screening and treatment, and partnering with policymakers for meaningful change.
• Maintaining a diverse, inclusive, safe, and healthy environment for its employees.

The company's commitment extends to conducting business in an environmentally responsible manner and continuously improving performance to benefit employees, customers, communities, and the environment.

As of December 2023:

• Patients in over 50 countries have received Ultragenyx treatments through commercial access or expanded use programs.
• More than 135 participants representing over 100 organizations have attended Ultragenyx's Rare Bootcamps since 2017.
• Ultragenyx has clinical trial sites in operation across 19 countries.

These figures highlight the global reach and impact of Ultragenyx's work in the rare disease community.

The company's core values are reflected in its culture, which promotes generosity, curiosity, inclusion, and humility. Ultragenyx fosters an environment that supports profound growth and fulfillment for its team members, who are passionate about helping those living with rare diseases.

Ultragenyx has several employee resource groups, including:

• UltraProud (LGBTQIA+ employees)
• Women in Biotech
• Ultra-Mosaic
• LatinX
• Ultra-APAC
• The Xtended Office
• Ultra Unique
• Ultra-Giving
• Ultra-Fun community group

These groups help drive awareness about important inclusion and diversity topics while inspiring positive change within Ultragenyx and beyond.

Ultragenyx Pharmaceutical Inc. (RARE) Vision Statement

A company's vision statement serves as a roadmap, outlining its aspirations and long-term goals. For Ultragenyx Pharmaceutical Inc. (RARE), understanding their vision provides valuable insight into their strategic direction and priorities within the biopharmaceutical industry. This chapter explores Ultragenyx's mission, vision, and core values, offering a comprehensive view of the company's guiding principles as of April 2025. To gain further insights into investor perspectives, explore Exploring Ultragenyx Pharmaceutical Inc. (RARE) Investor Profile: Who’s Buying and Why?

Ultragenyx's mission is to develop and deliver novel treatments for rare and ultra-rare genetic diseases. This involves:

• Identifying and acquiring promising drug candidates.
• Advancing these candidates through clinical development.
• Securing regulatory approvals.
• Commercializing therapies to reach patients in need.

The company focuses on diseases with significant unmet medical needs, often where no approved therapies exist. Ultragenyx's approach combines internal research and development efforts with strategic collaborations and acquisitions to expand its pipeline and expertise.

Ultragenyx envisions a future where innovative treatments transform the lives of patients with rare and ultra-rare genetic diseases. The company strives to be a global leader in the development and commercialization of therapies for these conditions. This vision encompasses:

• Pioneering new treatment approaches, including gene therapy, enzyme replacement therapy, and small molecule drugs.
• Building a sustainable business that can continue to invest in research and development.
• Collaborating with patient advocacy groups, researchers, and healthcare providers to improve the lives of those affected by rare diseases.

Ultragenyx's core values guide its employees and shape the company's culture. These values include:

• Patient Focus: Prioritizing the needs of patients with rare diseases in all decisions and actions.
• Innovation: Embracing creativity and scientific rigor to develop groundbreaking therapies.
• Collaboration: Working together effectively with internal teams and external partners to achieve common goals.
• Integrity: Upholding the highest ethical standards in all aspects of the business.
• Excellence: Striving for the highest quality in all endeavors, from research and development to commercialization.

These core values reflect Ultragenyx's commitment to making a meaningful difference in the lives of patients with rare genetic diseases. By adhering to these principles, the company aims to build a strong and sustainable organization that can continue to innovate and deliver life-changing therapies.

Ultragenyx Pharmaceutical Inc. (RARE) Core Values of

Ultragenyx Pharmaceutical Inc. is dedicated to developing treatments for rare and ultra-rare diseases. While specific, explicitly stated core values are not publicly detailed in the same manner as some companies, Ultragenyx's actions and communications reflect a strong commitment to several key principles.

These inferred core values are evident through their strategic initiatives, partnerships, and overall approach to addressing unmet medical needs. Here's an exploration of values that resonate strongly with Ultragenyx's operations:

Patient Focus

Ultragenyx places a strong emphasis on patients, particularly those with rare and ultra-rare diseases. This commitment is evident in their efforts to develop novel therapies that address significant unmet medical needs. The company's focus extends beyond just creating medications; they strive to improve the overall quality of life for patients and their families. For instance, Ultragenyx actively engages with patient advocacy groups to understand the challenges faced by the rare disease community and to ensure that their voices are heard. Ultragenyx’s commitment to patient focus is exemplified by:

• Developing therapies for diseases with very small patient populations, where traditional pharmaceutical companies may not invest.
• Providing patient support programs to help individuals access and adhere to their treatments.
• Collaborating with patient organizations to raise awareness and improve understanding of rare diseases.

Innovation

Innovation is central to Ultragenyx's approach to developing new treatments. The company invests heavily in research and development to identify and advance promising therapeutic candidates. This includes exploring novel technologies and approaches to address the underlying causes of rare diseases. Ultragenyx's dedication to innovation is reflected in its pipeline of diverse therapies, which includes:

• Gene therapies
• Enzyme replacement therapies
• Small molecule drugs

These innovative approaches aim to provide more effective and targeted treatments for patients with rare diseases. In 2024, Ultragenyx allocated a significant portion of its operating expenses, $643.9 million, to research and development activities, underscoring its commitment to innovation.

Collaboration

Ultragenyx actively seeks collaborations with academic institutions, other biotechnology companies, and patient advocacy groups to advance its mission. These partnerships enable the company to leverage external expertise and resources, accelerate the development of new therapies, and expand its reach to patients in need. Ultragenyx’s collaborative efforts include:

• Partnering with universities to conduct research on rare diseases and identify potential therapeutic targets.
• Collaborating with other companies to develop and commercialize new treatments.
• Working with patient advocacy groups to raise awareness, provide support, and advocate for policies that benefit the rare disease community.

Integrity and Ethics

Ultragenyx is committed to conducting its business with the highest standards of integrity and ethics. This includes ensuring the safety and well-being of patients participating in clinical trials, adhering to all applicable regulations, and maintaining transparency in its communications. Ultragenyx's commitment to integrity and ethics is demonstrated by:

• Implementing robust quality control measures to ensure the safety and efficacy of its products.
• Conducting clinical trials in accordance with ethical guidelines and regulatory requirements.
• Providing clear and accurate information to healthcare professionals and patients about its therapies.

Driving for Results

Ultragenyx demonstrates a clear drive for results, with a focus on achieving meaningful outcomes for patients and creating value for its stakeholders. This involves setting ambitious goals, executing efficiently, and continuously monitoring progress. As of the 2024 fiscal year, Ultragenyx reported total revenues of $460.5 million, reflecting the company's ability to generate significant revenue from its approved products. This financial performance underscores Ultragenyx's commitment to delivering value and achieving sustainable growth.

In summary, while Ultragenyx doesn't have a list of explicitly stated core values, their behaviors, strategies, and achievements point to a strong commitment to patient focus, innovation, collaboration, integrity, and driving for results. These inferred values guide the company in its mission to develop treatments for rare and ultra-rare diseases and make a positive impact on the lives of patients and their families.