Position Summary:

ultrafocused – Work together to fearlessly uncover new possibilities

The Senior Director, Medical Writing-Publications will provide strategic direction within the department and lead the successful development and tactical execution of publication plans across multiple programs in alignment with integrated evidence generation plans and lifecycle strategies and in compliance with SOPs, publication guidelines, and company goals. In addition, the Senior Director will be responsible for writing and coordinating the writing of peer-reviewed scientific publications, including distribution of work to medical writers or agencies as needed. They will serve as the primary contact for publication activities in collaboration with a cross‐functional group, including Clinical Development, Medical Affairs, Biometrics, Pharm/Tox, Research, and other relevant functions.  

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.

Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.

Responsibilities:

1. Lead key strategic initiatives within the department including publication strategy, planning, and execution of Ultragenyx products and pipeline publication plans, ensuring alignment with department and company goals. 
2. Provide leadership within the department by proposing and overseeing new initiatives and capabilities, such as establishing best practices for pubs strategy development and pull-through. 
3. Interact with external authors and key internal stakeholders to develop high-quality publications (manuscripts, abstracts, posters, slide presentations) for peer-reviewed journals and scientific congresses and provide publication support and ensure compliant development of company-sponsored publications. 
4. Ensure smooth and effective document management from start to finish (i.e., generation of a blank document to a final approved version), which may include, but is not limited to: first draft authoring, reference retrieval, generating bibliographies, editing (e.g., follow house style guide), and managing various processes (e.g., reviews, QC, QA, formatting, publishing, document archiving, journal and congress submissions).  
5. Liaise with relevant corporate functions and partner(s), as applicable, to ensure appropriate communication of publications‐related activities including manuscripts and congress abstracts/posters/presentations. 
6. Uses advanced communication and persuasion strategies to negotiate on behalf of their department or function on strategic matters. 
7. Contributes to strategic planning for the broader function and support high‐quality execution of Corporate‐wide initiatives. 
8. Acts as a trusted advisor and recognized technical expert. 
9. Manage and/or mentor junior medical writers and contractors. 
10. Support publication budget planning and management. 

Requirements:

1. BS, MS, or doctorate in a scientific or medical field. 
2. Solid experience (BS with >15 years or advanced degree with >10 years) in publication management and medical writing in the biotechnology/pharmaceutical industry preferred. 
3. Proven success with leading and managing medical writing agencies as well as track record for consistently meeting or exceeding quantitative targets. 
4. Demonstrated experience analyzing and communicating scientific data in peer-reviewed scientific publications; knowledge of applicable publications-related guidelines (i.e., ICMJE, GPP) and best practices, as well as legal, regulatory, compliance, and clinical/medical affairs. 
5. Experience writing and preparing manuscripts for publication in peer‐reviewed journals as well as abstracts, posters, and slide decks for presentation at scientific congresses. 
6. Successful and superior influencing skills across all levels of the organization and external collaborators. 
7. Exceptional oral and written communication skills. 
8. Experience in successfully engaging with cross‐functional product teams. 
9. Strong organizational abilities and experience in a multitasking environment. 
10. Motivated, self‐directed, and able to work autonomously and in team settings to meet aggressive organizational goals. 
11. Rare disease experience and a strong understanding of neurodevelopmental disorders are a plus. 

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Position Summary:

ultradedicated – Your biggest challenges yield rare possibilities

The Associate Director, GRA Global Labeling will be responsible for providing global labeling expertise and direction in support of the development, registration, and lifecycle management of innovative programs for rare diseases. This individual will liaise across key functional areas collecting critical regulatory milestones and developing detailed target label strategy plans during clinical development programs in order to align with company objectives and registration strategies. The responsibilities include managing labeling changes for lifecycle products, assisting in the development of labeling content for new products in pre-approval stages, and reviewing applicable regulatory documentation and artwork. The individual will also support implementation of approved labels which may include document control, QC/proofread, translations and verification of translations, and tracking project milestones and status. The individual must possess a thorough knowledge of global regulations and guidance governing drugs and biologics in all phases of clinical development. This position will report to Senior Director, GRA Global Labeling. 

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:

• Provides global labeling leadership in support of the development, registration, and life-cycle management of Ultragenyx products for rare diseases. 

• Prepare new and revised labeling content (Company Core Data Sheet, Prescribing Information, Summary of Product Characteristics, Major Market labels, Patient Information, and Instructions for Use) based on source reports, regulatory requirements, and other reference sources. 

• Demonstrate the ability to work in a matrix environment communicating label concepts and key messaging during label development consistent with program strategy and understanding and educating the impact to downstream activities (i.e., for commercial, and promotional educational purposes). 

• Build strong collaboration with key internal stakeholders, including Global Regulatory leadership team, Clinical Development, Safety, Quality, Commercial, Medical Affairs, Legal, Manufacturing, Supply Chain, and other functions to support development of draft label text.  

• Interact with the cross functional labeling team (Label Working Group and Label Review Committee) to ensure timely implementation of global labeling changes in local product information that are consistent with the company’s position defined in the CCDS and in compliance with local labeling regulations. 

• Prepare, deliver and/or manage all types of regulatory documents/submissions influencing and managing cross-functional team member input to provide high quality deliverables that meet regulatory standards, and corporate and program milestones. Facilitate LWG/LRC/SME meetings to develop content for label documents including resolution of complex label issues and ability to drive consensus utilizing current standard operating procedures, best practices and/or work instructions including work requests associated with partner companies and vendors. 

• Facilitate and lead target label strategies with cross functional labeling team incorporating key messaging aligned with target product profile and thorough understanding of competitive landscape environment. Provide necessary label output for HA interactions (i.e., FDA/EMA/PMDA/HC briefing book, scientific advice, and/or other key submission readiness milestones). 

• Manage approved labels in a document label management system to ensure accurate and up-to-date labeling is always available and accessible for global submissions and inspection readiness. 

• Contribute to the review and approval of artwork supporting global submissions. 

• Implement and maintain a quality control measure during all stages of label development. 

• Maintain knowledgebase of current global rules, regulations, and relevant guidance (draft or final) governing drugs and biologics in all phases of development, as well as knowledge of therapeutic area(s).   

• Initiate or contribute to local and/or global process and/or system improvements which have a significant impact on business. 

Requirements:

• Minimum BA/BS degree in life sciences. Advanced degree preferred. 

• Strong scientific background with 8 years’ experience in the biotechnology and/or pharmaceutical industry including at least 4+ years in a global regulatory/labeling position. 

• Excellent attention to detail with solid coordinating, task planning and time management skills. 

• Excellent verbal, written and interpersonal communication skills. 

• Ability to work independently and in a team environment, with strong leadership, negotiation and influencing ability. 

• Strategic thinker and problem-solver capable of providing advice, identifying risks, decision making in a collaborative way, and implementing plans and risk mitigation strategies. 

• Thorough understanding of global drug development regulations and guidelines. 
• Willingness to work in a dynamic, fast paced and changing corporate environment where roles and responsibilities are not well defined. 

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Position Summary:

Ultradedicated – Your biggest challenges yield rare possibilities

The National Field Leader will establish launch strategy for the field sales teams, performance drivers and the resourcing plans to support the Ultragenyx Neurology therapeutic area. This leader will possess broad experience in leading teams, business ownership, analytics, driving performance, and the implementation of innovative solutions. The National Field Leader will need to formulate an effective national go-to-market plan, while allowing territorial variations, including establishing both short- and long-term success factors and appropriate KPIs. It will be the responsibility of the National Field Leader to provide direction related to key objectives within the territories and implement a national field plan that is organizationally approved and fully compliant with all applicable laws and the company code of conduct.  

Work Model: 

Field: Officially documented as working as a member of the Ultragenyx field team, generally interacting with third parties on behalf of Ultragenyx. 

Responsibilities:

1. Build and lead a national field sales team consisting of multiple regions and territories in each region.  
2. Mentor and coach regional leaders to effectively manage their teams – providing constructive feedback and coaching to achieve superior performance and leading efforts in field sales employee development 
3. Set launch and growth objectives while aligning with the marketing strategy and priorities. Ensure the sales and marketing strategies, programs and tactics are uniformly adopted and implemented by the sales force members, within the parameters of the company’s operating values, culture, to achieve product sales market share and revenue goals.  
4. Operate in the best interest of patients and providers, ensuring a ‘patient-first’ focus, demonstrating the highest ethical and moral standards 
5. Ensure that the UltraCare Liaison (Rare Disease Field Sales) team is equipped & trained to address field market requests, customer service issues and challenges, with a sense of urgency and accountability  
6. Understand healthcare trends in the Rare Disease Community and create pull-through tactics and solutions to mitigate market access challenges  
7. Employ strong organizational and analytical skills to optimize growth, including the ability to analyze complex data to develop strategic and actionable business plans  
8. Develop and implement key sales team processes such as incentive plans, communication plans, recognition programs, performance management   
9. Conduct regular business and performance reviews for all direct reports and their team members, and effectively manage under-performers  
10. Lead and maintain a culture of compliance, integrity and transparency  

Requirements:

1. Minimum of bachelor's degree; Advanced degree highly preferable Minimum of 15+ years of experience in sales with at least 3 years in a leadership position in rare/ultra-rare disease driving growth businesses  
2. Lead with emotional intelligence while thriving in a rapid-paced and dynamic environment that aligns with Ultragenyx values.  
3. The National Field Leader will serve as a role model for the culture that relies on teamwork and collaboration using a blend of science and business skills and experience  
4. Strong business relationships in the Rare Neurology community with an in-depth understanding of the clinic multidisciplinary team environment 
5. Must have experience launching an ASO (antisense oligonucleotide) product
6. Motivational and inspirational leader who can affect change, collaborate well with other cross-functional leaders and executive-level leaders and demonstrated success in launching new therapies 
7. Experience building high performing teams, with a focus on culture, compliance and launch excellence  
8. Approximately 75% travel is required including overnight travel as needed  

#LI-AM1 #LI-Remote

Position Summary:

ultrafocused – Work together to fearlessly uncover new possibilities

We are seeking an experienced and highly motivated Senior Medical Science Liaison (MSL) to join our team. In this role, you will act as a key link between Ultragenyx and the rare disease medical community, providing critical scientific information about our neurology portfolio pipeline and prepare for commercialization of an anti-sense oligonucleotide for the treatment of Angelman syndrome. The Sr MSL will have a strong clinical and/or scientific background, exceptional communication skills and a passion to shape the future of rare disease medicine and will interact with KOLs and centers of excellence. The Sr. MSL will also build and maintain strong partnerships with cross-functional colleagues and work closely with the Field Medical leadership to ensure execution of the Field Medical plan.

Work Model: 

Field: Officially documented as working as a member of the Ultragenyx field team, generally interacting with third parties on behalf of Ultragenyx

Responsibilities:

· 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.

Requirements:

· Terminal degree (e.g., MD, PhD, PharmD) in a clinical or medical specialty and minimum of 2 years prior experience as an MSL

· Candidates with an advanced degree (e.g., MS, MSN, MPH) and at least 5 years MSL experience in a related field to the therapeutic area will also be considered

· Demonstrated technical and scientific expertise in rare disease is preferred

· Demonstrated excellence in delivering high-impact clinical and scientific presentations to expert audiences in both virtual and live settings.

· Exceptional understanding of the health care delivery system and its impact on patient care and regional medical care

· Knowledge in interpreting and adhering to FDA regulations regarding product promotion, scientific exchange, and health care practitioner guidelines

· Thinks strategically and leverages knowledge of general business practices, the industry, medical practices and Ultragenyx strategic objectives to identify field opportunities

· Ability to relate and work with a wide range of people to achieve results

· Ability to work autonomously to organize and prioritize one’s own work schedule

· Ability to build and leverages strategic internal and external relationships to identify potential opportunities and partnerships to provide value and improve quality of care for patients

· Accountable for individual territory management

· Proactively and consistently delivers on commitments, prioritizes time, actions, and resources to meet goals

· Approximately 60% travel in region and nationally is required; overnight travel is required as needed

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Position Summary:

ultracurious – Apply your biggest ideas in courageous ways

The Director of Disease Monitoring Programs (DMPs) is a visible leadership role accountable for the strategic planning and oversight for one or more assigned DMPs.  DMPs are a novel approach to GCP-compliant long-term data collection for rare diseases from pre-approval through post marketing. The Director of DMPs will collaborate with other functional leaders at both tactical and strategic levels.   

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:

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

Requirements:

1. Bachelor degree or equivalent required (scientific or healthcare discipline preferred) 
2. 10+ years of progressively challenging experience in clinical research and/or drug development 
3. Critical thinking skills coupled with innovative approaches to problem solving 
4. Comfortable managing and presenting to senior level stakeholders 
5. Comprehensive understanding of high-quality data generation appropriate for regulatory, scientific, payer, physician and patient requirements to advance the understanding of rare diseases and their treatment/management  
6. Ability to provide operational expertise to a clinical development program 
7. Working knowledge of regulatory agency regulations and ICH GCP guidelines is required 
8. Strong strategic planning and decision-making skills 
9. Excellent written and verbal skills required.  Must display strong analytical and problem solving skills   
10. Willing and able to travel domestically and internationally 
11. Rare disease clinical trial experience desired 

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Position Summary:

ultraimpact – Make a difference for those who need it most

We are looking for experienced UltraCare Liaisons (UCLs) that will represent Ultragenyx in the Rare Disease Community, responsible for the promotion of Dojolvi for the treatment of long chain fatty oxidation disorders (LC-FAOD). Ultragenyx is also preparing for the potential commercialization of two investigational novel gene therapies in MPSIIIA and GSD1a. We are seeking driven individuals with the desire to run their own business within a specified geography. These individuals will have strong business acumen, be able to drive change through innovative approaches, demonstrate excellent communication skills and consistently deliver sales results.

Work Model: 

Field: Officially documented as working as a member of the Ultragenyx field team, generally interacting with third parties on behalf of Ultragenyx. 

Responsibilities:

1. Serve as territory business owner with a focus upon impact and territory analysis. Other key areas of focus include optimizing: i) patient diagnosis and care through HCP education, ii) educating HCPs about Ultragenyx approved products, iii) assisting with treatment fulfillment
2. Ability to develop and nurture effective business relationship management with key stakeholders, including HCPs, Registered Dieticians, Genetic Counselors, Pharmacists, and related support staff
3. Proactively builds effective working relationships with internal/external stakeholders; can drive agreement/decisions from multiple stakeholders; ability to understand people’s emotions and flex communication style. Can adjust their approach based on different stakeholder needs, concerns, or audience member to drive alignment and meet their work goals.
4. Develop and maintain a strategic territory business plan focused on key academic centers, community targets within priority specialties, territory opportunities and challenges.
5. Determines and implements suitable travel schedule and call plan on a daily/weekly basis to ensure both adequate and highly effective coverage for all key accounts.
6. Executes programs, high impact in-services, and other educational opportunities for their territory.
7. Timely completion of compliance trainings, internal product & disease state trainings, Veeva administration, monthly expense reports, and all other administration expectations.

Requirements:

1. Bachelor’s Degree required
2. 8 years with 5+ years being in the healthcare/biotech industry, inclusive of 3 years of field-based experience in account management, sales, and/or field reimbursement. Rare disease experience is preferred.
3. Experience launching biopharma/pharma products successfully is preferred
4. Documented track record of field sales success
5. Strategic business acumen and cross-functional and collaborative leadership with internal stakeholders including marketing, medical, clinical operations, and patient services.
6. Demonstrated experience effectively presenting clinical/scientific information required
7. Approximately 50 - 60% (dependent on geography) travel is required; overnight travel is required as needed
8. Must live in territory geography. Territory includes: Washington, Oregon, Idaho, Montana, Wyoming, Northern California. Territory subject to change based on business need

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Position Summary:

ultradedicated – Your biggest challenges yield rare possibilities

Ultragenyx is seeking a dynamic and experienced physician to join the Gene Therapy Treatment Team. The physician will play a key medical affairs leadership role within the gene therapy portfolio, initially focused on GSDIa and MPS IIIA, where the company is in position to launch two gene therapies this year. This position will be instrumental in providing clinical and scientific education to gene therapy treating centers to ensure that patients receive their one-time therapy in the most effective manner and achieve their optimal therapeutic outcome.  

Reporting to the VP Global Medical Expert – Gene Therapies, the Executive Director is a senior medical affairs leader responsible for ensuring scientifically rigorous, compliant, and effective implementation of one-time gene therapies. The Executive Director will work closely with Medical Science Liaisons in the high-profile and innovative Gene Therapy Treatment Team through a largely externally facing role. The Executive Director also partners closely with Medical Affairs, Clinical Development, Regulatory, and Commercial leadership to enable successful launch and post launch adoption, while shaping external scientific dialogue around safety, durability, immunomodulation, and patient outcomes.   

The role requires extensive peer-to-peer interactions with external physicians providing gene therapy treatment in the commercial setting. The successful candidate will be comfortable working in a fast-paced intellectual environment, understand patient care including immunomodulation, and is able to work at the interface between clinical development and medical affairs leveraging their expertise to advance our mission to change the future of rare disease medicine. 

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:

• Serve as a senior external medical leader for Ultragenyx gene therapies, leading compliant complex, scientific discussions with treating physicians  

• Enable successful implementation of gene therapies globally both within Ultragenyx and with external medical experts 

• Respond to inquiries and questions from treating sites with appropriate level of urgency 

• Lead the strategic and scientific requirements for educational materials and lead the delivery of training for the gene therapy treatment centers to ensure those centers are well prepared to manage patients throughout their gene therapy treatment journey 

• Provide expertise and contribute as needed to protocol development, steering committee meetings, data generation initiatives of Disease Monitoring Programs, and life-cycle management for label/indication expansion 

• Advise on strategic development of publications and medical materials that enable external medical engagement and address evidence gaps to inform clinical decision making 

• Work in partnership with VP, GME – Gene Therapies to develop and implement the global Gene Therapies Treatment Team Framework  

• Mentor and develop Global Medical Expert physicians and MSLs, helping build a scalable, high‑performing medical organization to support current and future gene therapy launches 

• Provide expertise and contribute as needed to key regulatory agency communications, including filings and information request responses 

• Represent Ultragenyx at key scientific and external stakeholder meetings to advance awareness and deep understanding of our science and therapies 

• As an expert, lead the education and training of Ultragenyx internal teams  

• Stay abreast of internal and external developments, trends and dynamics and provide direction for strategic implications to the development and commercialization of gene therapy  

Requirements:

• Medical Doctor (MD required, MD plus PhD preferred) with Board Certification (or equivalent) in Clinical Immunology, Clinical/Biochemical Genetics, Rheumatology or Neurology (required) 

• Academic/Faculty experience (+7 years) with a track record of high-quality publications   

• Pharmaceutical experience in drug development and clinical trials or Medical Affairs (+7 years) 

• Experience in the management of patients receiving systemic AAV gene therapy in the clinical trial and/or post FDA approval setting  

• Demonstrated ability to prioritize and lead complex post‑approval gene therapy activities, applying sound scientific judgment while enabling cross‑functional decision‑making 

• Demonstrated ability to lead through ambiguity and make strategic medical decisions in first‑ or best‑in‑class therapeutic settings 

• Availability for some out-of-hours work is an expectation 

• Demonstrated success working in a cross functional or multidisciplinary setting is essential 

• An established broad network of key experts and leaders within the rare disease community  
• Travel: 25-50% but may reduce with time as launches progress and team grows   

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