Ultragenyx

• Lead in product / indication level tasks including regulatory interactions and filing, and ensure statistical integrity; contribute strategically to the supporting projects from statistics perspective • Contribute to study level tasks from statistics perspective, including: study design and sample size determination; Author/review statistics section in the protocol, SAP and DMC charter; Create/review study randomization files; Develop TFL shell and specification Review CRFs and other study documentations; Active participation in study related meetings • Works collaboratively within biometrics teams and with cross-functional teams to meet product deliverables and timelines for statistical data analysis and reporting • Ensure statistical integrity of deliverables; provide statistically sound scientific methodology input to meet project objectives and regulatory statistical and data requirements • Independently conduct analyses suggested by the data; Propose new/novel statistical methodological approaches to improve the efficiency and sensitivity of study results • Contribute to developing standards and research in advanced statistical methodologies • Author/review regulatory documents or scientific publications • Mentor junior team members

• Learn and track regulatory approval pathways for all cases; ensure approvals are collected, up to date, and compliant. • Maintain audit-ready documentation in accordance with industry standards. • File study documents in designated systems (e.g., electronic Trial Master File), maintain essential document lists, and participate in TMF completeness reviews. • Initiate and maintain MA/IST contracts and amendments. • Coordinate drug supply requests and collaborate with distribution vendors and the vendor manager to monitor shipments, resolve issues, and ensure uninterrupted supply. • Proactively identify risks and collaborate with peers and senior team members to mitigate them. • Adhere to departmental processes and high standards of organization and documentation. • Manages case monitoring activities ensuring compliance with Good Clinical Practices (GCP) and applicable regional/country specific regulations.

• Identify, establish and maintain long-term, strategic scientific relationships with KOLs and proactively liaise appropriately with internal stakeholders ensuring research, scientific, and clinical interests are communicated • Provide fair balanced presentations and medical information in response to unsolicited questions as appropriate in academic, community, and healthcare provider settings • Develop and execute US territory and KOL engagement plans to enable high-quality, impactful scientific engagement and education. • Gather and lead reporting of medical and clinical insights that contribute to enhancement of Ultragenyx’s key medical positions, plans, future research and development efforts. • Analyzes KOL’s scientific point of view on Ultragenyx’s medical and scientific positions on rare diseases and executes appropriate action plan • Provide field-based medical affairs assistance for clinical research initiatives supported by the company, related to Investigator Initiated Trials, or other collaborative research and initiatives • Collaborate with the Clinical Operations organization to enhance patient enrollment in company-sponsored clinical trials by identifying appropriate clinical trial sites and interacting with investigators in ongoing studies • Leads projects/initiatives internally and represents medical affairs cross-functionally • Represent Ultragenyx at scientific conferences and meetings, engaging with KOLs and collating scientific data insights. Provide leadership to plan impactful medical engagement activities and deliver timely post-congress insight and competitive intelligence reporting. • Partner cross-functionally in assigned region, to optimize customer experience • Ensures all activities are compliant with company policy and procedures, which includes documentation of all relevant MSL activities in CRM platform, management of travel and expense budget, and accurate and timely completion of administrative reports, projects, and required training.

• Process optimization and execution in providing real world evidence to key stakeholders including patients, providers, researchers, and patient advocacy groups • Ensure evidence strategy alignment throughout the development cycle for a program and across functions • Implement best practices for one or more assigned DMPs to monitor key deliverables (e.g. evidence generation, achievement of goals, financial health) • Provide input on the development of protocols, goals and endpoints tables, case report forms, IEPs, publications, Steering Committees and other relevant deliverables • Represent the DMP S&M function on the project teams, such as the Development Sub-Team, Core Teams (as applicable), etc • Train and mentor cross-functional personnel working on DMPs as needed

• Early engagement planning: Define and execute sourcing strategies aligned to pipeline, portfolio priorities, and enterprise objectives. • Vendor selection: Lead RFIs/RFPs, competitive bidding, vendor evaluations, bid defense strategy, and onboarding alignment for complex and high-impact initiatives to support fit-for-purpose vendor selection and successful engagement start-up. • Contracting alignment: Lead alignment across scope, budget, and contracting considerations, ensuring consistency and scalability between operational requirements, financial assumptions, and vendor agreements. • Strategic vendor partnership management: Serve as a senior strategic partnership lead for external GCP vendors across service categories, driving partnership alignment, performance expectations, strategic engagement, and ongoing relationship management across the vendor partnership. • Governance & oversight: Lead governance forums and support timely escalation resolution through appropriate senior stakeholder engagement, while driving KPI/performance discussions, quality alignment, consistent oversight, accountability, and strategic alignment across partners. • Sourcing and partnership optimization: Drive complex cross-program sourcing initiatives, vendor/category alignment activities, and partnership optimization efforts to establish consistency of sourcing and partnership approach, strengthen vendor engagement, and scale effective practices across programs and vendors. • Lessons learned / continuous improvement: Drive continuous improvement by identifying cross-program insights, scaling best practices, and strengthening sourcing and partnership approaches across the portfolio. • Stakeholder engagement: Engage and influence senior cross-functional stakeholders to support sourcing decisions, align vendor partnership strategy with execution needs, and drive strategic and portfolio alignment across programs and vendors.

• Provide leadership and management to a National Field Team of Patient Access Liaisons (PALs). • Establish performance and development goals for PALs and monitor their progress throughout the year • Identify Access & Reimbursement trends and collaborate with cross functional team to address issues and in a timely manner and properly align resources (ROAR) • Ensure all Patient Access Liaisons are capturing all relevant data correctly in Patient Relationship Management System • Provide reporting metrics on a consistent basis in order to ensure all cases are moving along in a timely fashion • Organize and lead weekly team meetings with PALs and weekly meetings with counterparts that lead the Patient Engagement Centers • Monitor program processes to ensure there is efficiency and there is best in class customer experience • Create documents such as standard operating procedures, work instructions, training decks, etc. • Monitor all PALs reimbursement conflict resolution with their assigned accounts • Work cross functionally with internal and external stakeholders on any future franchise products

Title: Associate Director, Gene Therapy Marketing - Japan Location: Tokyo, Japan Job Description: At Ultragenyx, we fundamentally believe that taking real impactful action to care for the needs of patients and our people is always the right thing to do. To achieve this goal, our vision is to lead the future of rare disease medicine. For us, this means going where other biopharma companies won’t go – challenging the status quo and creating a new model that advances our field so more patients and caregivers can benefit from life-changing treatments. We do this by following the science, applying a novel rapid development approach, making innovative medicines at fair and reasonable prices, and creating a collaborative ecosystem to reach patients in ways that are most meaningful for them. Our commitment and care for patients extends to our people, so culture is an essential cornerstone for Ultragenyx. We remain continuously focused on creating a supportive and inclusive environment of profound learning and growth – so employees can thrive in all areas of their lives, in and outside of work. Ultimately, we want to be an organization where we would be proud for our family, friends and children to work. If you want to have a meaningful impact, do the best work of your career, and grow a lot, both professionally and personally, come join our team. Position Summary: ultraimpact – Make a difference for those who need it most Reporting to the Senior Director Gene Therapy Marketing, the successful candidate will lead marketing initiatives for a gene therapy product in collaboration with the Japan team and Global team. The role will involve the planning, development and execution of best-in-class annual marketing plans that are based on deep marketing knowledge and previously successful rare disease & biopharmaceutical marketing experience. This role will require developing and expanding the national network of Qualified Treatment Centers (QTCs) for gene therapy. This role oversees site identification, qualification, contracting, and readiness activities. As a growing and entrepreneurial organization, the candidate will participate in other business activities to support the growth of the company.   Work Model: Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed. Responsibilities: - Work closely with the Global marketing teams to lead the development of Japanese marketing initiatives, monitor the progress of these marketing initiatives and adjust appropriately to optimize success and ensure achievement of business and sales objectives for gene therapy. Ensure all marketing programs are adherent to Ultragenyx policies, procedures and compliance procedures  - Executing Marketing advisory boards and other HCP engagements to support market understanding and inform strategic and tactical planning and execution  - Building and developing meaningful business relationships with HCPs and with necessary external stakeholders across Japan  - Monitor and work to adapt the patient support program and its offerings to ensure optimal patient experience and product usage  - Manage congress initiatives in collaboration with the event management team, including messaging, booth design and coordinating onsite materials and field training.  - Identify issues that could potentially create barriers to optimal patient management and provide solutions  - Work cross-functionally and partner with multiple non-commercial teams including Global Brand Strategy, Government Affairs, Medical Affairs, Regulatory Affairs, & Patient Advocacy  - Partner with commercial colleagues and the customer facing Field Teams, providing training and ensuring field teams are appropriately prepared to deploy marketing programs   - Collaborate with Medical and Commercial Field teams to identify and assess prospective QTCs and manage the contracting process, including in person site visits as needed. - Oversee coordination and progress of QTC readiness activities, vetting, contracting, and POD team engagement and ensure timely site activation. Requirements: - Strong passion to deliver innovative new drugs to patients who are suffering from rare or ultra-rare diseases - BA/BSc degree or equivalent life-sciences qualification required. MBA preferred - Minimum 3+ years of marketing experience and 3+ Years sales experience in the biopharmaceutical industry with preference to those with experience in rare/ultra-rare disease. Gene therapy experiences are preferred. - Comfort working in start-up environments and working to define process and forge new operating principles with internal stakeholders  - Successful candidates will have a proven ability to work in a multidisciplinary team with a high degree of collaboration and a strong patient focus  - Strong command of analytical and market research techniques with a proven ability to interpret market insights to help guide strategic direction and prioritize patient tactics  - Experience of leading or collaborating on Market Access initiatives to expand patient access  - Experience of marketing Advisory Boards is preferred   - Demonstrated ability to think strategically and act creatively to develop and lead innovative brand strategies and tactics  - Proven experience in partnering with Field Teams  - Proven experience working with and managing external partners, including Advertising and Communications agencies  - Native-level proficiency in Japanese, as well as excellent public presentation skills, written communication and listening skills in English - Travel will be frequently required #LI-CK1 #LI-Remote Full Time employees across the globe enjoy a range of benefits, including, but not limited to: · Generous vacation time and public holidays observed by the company · Volunteer days · Long term incentive and Employee stock purchase plans or equivalent offerings · Employee wellbeing benefits · Fitness reimbursement · Tuition sponsoring · Professional development plans * Benefits vary by region and country Ultragenyx Pharmaceutical is an equal opportunity employer and prohibits unlawful discrimination based on race, color, religion, gender, sexual orientation, gender identity/expression, national origin/ancestry, age, disability, marital and veteran status, and any other status or classification protected by applicable federal, state, and/or local laws. Reasonable accommodation will be provided for all protected statuses or classifications protected by applicable law, including individuals with disabilities, disabled veterans, for pregnancy, childbirth, and related medical conditions, and based on sincerely held religious beliefs. Applicants can request an accommodation prior to accepting a job offer. It is unlawful in Massachusetts to require or administer a lie detector test as a condition of employment or continued employment. An employer who violates this law shall be subject to criminal penalties and civil liability. Note to External Recruiters: All candidate activity and open positions are managed strictly through our Human Resources Department. Our Human Resources Department kindly requests that recruiters not contact employees/hiring managers directly in an attempt to solicit business and present candidates. Please note that failure to comply with this request will be a factor in determining a professional relationship with our organization. Submission of unsolicited resumes prior to an agreement set in place between the Human Resources Department and the recruiting agency will not create any implied obligation.

Title: Director External Quality Assurance Location: Novato, CA; South San Francisco, CA Job Description: Be a hero for our rare disease patients At Ultragenyx, we fundamentally believe that taking real impactful action to care for the needs of patients and our people is always the right thing to do. To achieve this goal, our vision is to lead the future of rare disease medicine. For us, this means going where other biopharma companies won’t go – challenging the status quo and creating a new model that advances our field so more patients and caregivers can benefit from life-changing treatments. We do this by following the science, applying a novel rapid development approach, making innovative medicines at fair and reasonable prices, and creating a collaborative ecosystem to reach patients in ways that are most meaningful for them. Our commitment and care for patients extends to our people, so culture is an essential cornerstone for Ultragenyx. We remain continuously focused on creating a supportive and inclusive environment of profound learning and growth – so employees can thrive in all areas of their lives, in and outside of work. Ultimately, we want to be an organization where we would be proud for our family, friends and children to work. If you want to have a meaningful impact, do the best work of your career, and grow a lot, both professionally and personally, come join our team. Position Summary: ultrafocused – Work together to fearlessly uncover new possibilities The Director, CMC QA will report to the Senior Director of CMC QA and be responsible for maintaining strategic responsibility over QA Manufacturing Operations, developing and implementing Ultragenyx CMC Quality approach for assigned programs, and working with colleagues to improve processes utilized to ensure product quality. In addition, this position will use strong management skills to independently manage external partnerships. In this role, in collaboration with key stakeholders in Technical Operations, DIR CMC QA provides quality oversight and support to ensure clinical through commercial development lifecycle activities for drug substance and drug product meet cGMP regulatory requirements and guidelines. Work Model: Flex: This role will typically require onsite work 2-3 days each week, or more depending on business needs. In many locations, the business will set certain days each week that Flex employees are required to be onsite. Responsibilities: - Review/approve investigation of operations in manufacturing/laboratory or other related operations and lead the strategic discussion for investigations with respect to operations related matters. Oversee resolution of quality events, monitor that recurrent events are properly escalated and resolved. Ensure root cause is determined, evaluate impact on product quality, disposition, and corrective actions. - Review and approve changes related to facility, computerized systems, laboratory, or products. - Perform final review of test data/reports to ensure conformance to the established specifications and standard operating procedures. - Drive execution of risk assessments related to manufacturing processes for small molecule, biological products, and novel therapeutics (i.e., mRNA). - Manage the lifecycle of products within the portfolio according to quality standards. - Proactively engage with Tech Ops in addressing qualification requests. - Proactively engage with Tech Development in addressing process development requests. - Develop and maintain procedures and practices in compliance with FDA, EMA, and local regulations and guidance, ICH guidelines, Ultragenyx policies/procedures, and industry best practices. - Other duties as assigned. Requirements: - Bachelor’s degree preferably in an Engineering, Biotechnology, Biological/Physical sciences, or equivalent. - 10+ years of experience in a GMP biopharmaceutical manufacturing environment in technical operations and/or quality. - Strong working knowledge and interpretation of FDA, EMA, local regulations and guidelines, Good Distribution Practices, ICH guidelines and industry best practice. - Experience using and facilitating use of risk management tools (FMEA, PHA, etc.) - Experience with performing technical assessment of changes for potential impact to qualification/validation of processes, equipment, utilities, facilities, and systems. - Capability of collaboratively engaging with Contract Organizations. - Experienced in all phases of biologic and small molecule drug development. - Demonstrated high personal and professional ethical standards. - Possesses excellent judgment with the ability to think and act quickly. Must be able to identify creative solutions to complex technical problems. - Ability to interpret and relate Quality standards for implementation and review. - Ability to communicate clearly and professionally both in writing and verbally. - Strong organizational and project management skills with a track record of meeting goals/objectives. - Strong experience with regulatory authority inspections, including both FDA and EMA. - Travel will be required when appropriate (approximately 10%) #LI-CS1 #LI-Hybrid The typical annual salary range for this full-time position is listed below. This range reflects the characteristics of the job, such as required skills and qualifications and is based on the office location noted in this job posting. The range may also be adjusted based on applicant's geographic location. This position is eligible for annual bonus and equity incentives. Actual individual pay is determined by demonstrated experience and internal equity alignment. Pay Range $215,600 - $266,300 USD Full Time employees across the globe enjoy a range of benefits, including, but not limited to: · Generous vacation time and public holidays observed by the company · Volunteer days · Long term incentive and Employee stock purchase plans or equivalent offerings · Employee wellbeing benefits · Fitness reimbursement · Tuition sponsoring · Professional development plans * Benefits vary by region and country Ultragenyx Pharmaceutical is an equal opportunity employer and prohibits unlawful discrimination based on race, color, religion, gender, sexual orientation, gender identity/expression, national origin/ancestry, age, disability, marital and veteran status, and any other status or classification protected by applicable federal, state, and/or local laws. Reasonable accommodation will be provided for all protected statuses or classifications protected by applicable law, including individuals with disabilities, disabled veterans, for pregnancy, childbirth, and related medical conditions, and based on sincerely held religious beliefs.

• Define and lead the framework, standards, and operating model for integrated evidence generation, including processes and cross-functional ways of working, to enable consistent, high-quality development and execution of Integrated Evidence Plans (IEPs) across programs • Partner with Global Medical Leads and cross-functional teams to support development of IEPs and align on medical evidence generation approaches • Translate IEP priorities into execution-ready evidence generation plans, including study concepts, protocols, data sourcing strategies, analytical approaches, and input into Disease Monitoring Programs (DMPs) • Lead and coordinate evidence generation activities (e.g., RWE studies, registries, chart review studies, natural history studies) and contribute to DMP strategy and design, in collaboration with cross-functional stakeholders, clarifying roles and responsibilities for execution as operating models evolve • Serve as an internal subject matter expert on real-world data (RWD) and real-world evidence (RWE), providing strategic guidance on data source selection, methodological approaches, and application of RWD/RWE to inform evidence generation and decision-making • Establish and advance structured data planning approaches and enable selection of fit-for-purpose data sources (e.g., EMR, claims, registries, PROs, and DMP-derived data), ensuring consistency, quality, and utility of data across programs • Lead and guide the analysis, interpretation, and dissemination of data generated from prioritized evidence generation activities, including DMP outputs, to support publications, scientific communications, and post-marketing and real-world evidence activities supporting regulatory, payer, and clinical decision-making • Implement and evolve enterprise-level processes and KPIs to support prioritization, tracking, and delivery of evidence generation activities, and partner cross-functionally (Clinical, HEOR, Regulatory, Market Access) and with external researchers and academic partners to ensure alignment with IEP priorities and evidence generation objectives; participate as a subject matter expert in IST/IIR/CR review processes

• Day-to-day administrative support for the Canadian Vice President & General Manager, and functional leads in Medical Affairs, Market Access, Commercial Operations, Marketing, as well as support for Canadian field teams • Proactively manage calendar, appointments, and meeting requests through MS Outlook • Communicate with Canadian leadership, external partners, and internal stakeholders to schedule and set up in-person and virtual meetings • Participate in special projects and project management work as required by the business • Contract coordination — support contract-related administrative tasks including document preparation, routing for signatures, and maintaining organized contract files • Compliantly manage financial processes including purchase order creation and management, invoice payment, budget management and monitoring, and other necessary processes • Contract creation, review support, lifecycle management, and tracking across internal systems • Monitor contract timelines, ensuring renewals, expirations, and compliance requirements are proactively addressed • Track invoices and honoraria and ensure timely payment • Develop tracking mechanisms in Excel for external expense budgets in alignment with invoices • Ensure adherence to compliance requirements for payment of honoraria and invoices as well as vendor contract management • Manage and access necessary online portals/software (iCertis, Oracle, Veeva, OneDrive, Adaptive, SharePoint, Concur, Zoom, Microsoft Teams, etc.) and others as needed • Conduct regular check-ins with other administrative personnel to optimize activity • Coordinate interviews, onboarding, and hiring processes