Director, Regulatory CMC - Gene Therapy

Ultragenyx Pharmaceuticals Cambridge , MA 02138

Posted 1 week ago

Why Join Us?

Be a hero for our rare disease patients

Ultragenyx is a commercial-stage biopharmaceutical company committed to developing novel therapies for the treatment of rare and ultra-rare genetic diseases. The company has rapidly built a diverse portfolio of small molecule, monoclonal antibody, mRNA and gene therapy candidates with the potential to address debilitating genetic diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are typically no approved therapies treating the underlying disease.

With the company's recent acquisition of Dimension Therapeutics, the Ultragenyx Gene Therapy division was established. The Ultragenyx Gene Therapy division is headquartered in Cambridge, MA. We are looking for exceptional individuals who share our passion for developing novel therapies to treat rare diseases and advancing the field of gene therapy.

Developing new treatments for rare genetic diseases is challenging work. It takes courage, talent, and dedication to look past the easy answers and uncover new solutions that can make a meaningful impact. At Ultragenyx we push each other to perform at our very best, because we never lose sight of our mission - to make a difference in our patients' lives.

Come join our team during this exciting time of growth and opportunities! During the COVID-19 Outbreak we are committed to our hiring process. The health and safety of our employees and prospective employees take priority which means at this time our process will include remote based web-ex video discussions, interviews & onboarding.

Position Summary:

The Senior Director, Regulatory CMC will be responsible for leading and managing the regulatory affairs CMC function. This includes developing and leading regulatory CMC strategies and activities for the organization on a global basis. The Senior Director will ensure that all CMC regulatory goals are clearly defined and they align with current industry standards and regulatory requirements. This role will also provide significant input on applicable regulatory affairs processes, procedures, and tools to facilitate effective execution by the regulatory function across the organization. This individual must be highly skilled and provide expertise in CMC agency interactions and developing regional regulatory CMC strategies. In addition the position assists corporate senior management in understanding CMC Regulatory strategies/processes while developing corporate objectives and, importantly, understanding the impact of decisions across therapeutic areas.

Position Reporting To:

Vice President, Global Head of Regulatory Affairs

Responsibilites:

  • Develop the CMC regulatory strategies and manage related regulatory activities. Work with the Pharmaceutical Development team to prepare project plans and timelines for pipeline of products, including coordination and oversight of all types of regulatory CMC submissions.

  • Supervises/manages others to ensure that all reporting requirements and other commitments are met with respect to INDs, BLAs, CTA, MAAs, etc

  • Advise the global regulatory compliance of Ultragenyx, Gene Therapy's pipeline of products including the evaluation of CMC related change controls, the strategic assessment of process and product comparability, their regulatory impact and implementation.

  • Lead the preparation, review and filing of CMC sections of regulatory submissions and interactions with regulatory agencies.

  • Oversee, coordinate and ensure the preparation of regulatory CMC submission documentation is prepared with quality and in a timely manner to meet all agency requirements and corporate objectives.

  • Maintain a centralized archive for tracking and monitoring the status of regulatory CMC commitments and agency reporting obligations. Create and issue status reports and other communication to the organization.

  • Provide strategic CMC regulatory support, guidance and expertise, and act as the Regulatory Affairs primary point of contact to cross-functional teams including Pharmaceutical Development, Quality Assurance, Quality Control, contract organizations and consultants. Ensure that all applicable regulatory requirements/options and associated risks are considered and appropriately incorporated into development programs and that products are developed and manufactured in compliance with appropriate regulations and guidelines.

  • Work across functional groups in identifying issues that may delay timelines, providing solutions to difficult technical/functional issues associated with specific projects, as well as support the resolution of issues that arise.

  • Insure the regulatory conformance of technical documentation including technical transfer protocols, validation protocols, technical reports, manufacturing and labeling information.

  • Provide guidance and advice on regulatory CMC environments; provide assessment of the impact of new and changing regulations/requirements.

  • Support other Regulatory Affairs functions as needed.

Qualifications:

  • BA/BS in a scientific field of study with 12 years + of relevant experience working in Regulatory in the pharmaceutical/ biotech industry.

  • Strong knowledge, experience and leadership in interpretation of regulations and guidelines related to biologics drug development.

  • Experience in the Gene Therapy or Rare Disease area preferred.

  • Proven ability to analyze and organize information logically.

  • In depth understanding and application of industry standards and international regulations and guidelines.

  • Experience in communicating regulatory strategy, submission documents and plans both internally and externally.

  • The ability to coordinate and prioritize multiple projects in a fast-paced, deadline driven start-up environment.

  • Strong collaboration, team work, organizational skills at attention to detail

  • Excellent written and verbal communication skills.

  • Travel 20%

#LI-CZ1

Ultragenyx Pharmaceutical is an equal opportunity employer & prohibits unlawful discrimination based on race, color, religion, gender, sexual orientation, gender identity/expression, national origin/ancestry, age, disability, marital and veteran status. Reasonable accommodation will be provided for qualified individuals with disabilities and for qualified disabled veterans in job application procedures, as required by applicable law. If you have any difficulty using our online application system and need an accommodation due to a disability, you may contact Talent Acquisition by calling: (415) 483-8800 or by emailing us at talentacquisition@ultragenyx.com. For electronic email and mail inquiries, please include a description of your requested accommodation, your name and contact information.

See our CCPA Employee and Applicant Privacy Notice.

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. Inquiries on developing a recruiting relationship with us, may be directed to: talentacquisition@ultragenyx.com.


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Senior Director Regulatory Affairs (Clinical/Nonclinical Strategy) Gene Therapy

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VIEW JOBS 3/20/2020 12:00:00 AM 2020-06-18T00:00 Why Join Us? Be a hero for our rare disease patients Ultragenyx is a commercial-stage biopharmaceutical company committed to developing novel therapies for the treatment of rare and ultra-rare genetic diseases. The company has rapidly built a diverse portfolio of small molecule, monoclonal antibody, mRNA and gene therapy candidates with the potential to address debilitating genetic diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are typically no approved therapies treating the underlying disease. With the company's recent acquisition of Dimension Therapeutics, the Ultragenyx Gene Therapy division was established. The Ultragenyx Gene Therapy division is headquartered in Cambridge, MA. We are looking for exceptional individuals who share our passion for developing novel therapies to treat rare diseases and advancing the field of gene therapy. Developing new treatments for rare genetic diseases is challenging work. It takes courage, talent, and dedication to look past the easy answers and uncover new solutions that can make a meaningful impact. At Ultragenyx we push each other to perform at our very best, because we never lose sight of our mission - to make a difference in our patients' lives. Come join our team during this exciting time of growth and opportunities! During the COVID-19 Outbreak we are committed to our hiring process. The health and safety of our employees and prospective employees take priority which means at this time our process will include remote based web-ex video discussions, interviews & onboarding. Position Summary Ultragenyx is seeking an experienced and highly motivated Sr. Director, Regulatory Affairs (Clinical/Nonclinical Strategy), who is a team builder and enjoys a fast paced, dynamic work environment. The Sr. Director will be responsible for providing global regulatory leadership in support of the development, registration, and life-cycle management of our innovative gene therapy programs, as well as for the oversight and mentoring of a growing regulatory team. The responsibilities include providing leadership and direction for the global regulatory aspects of the gene therapy products, including developing long and short-term planning of regulatory projects that align with the company's business plan, and developing strategies to ensure effective achievement of regulatory/business objectives. The individual will be responsible for oversite and preparation as needed for multiple regulatory documents and collaboration with outside regulatory agencies, corporate partners and vendors in regards to development, regulatory, and registration strategies. The individual will also maintain awareness of the global regulatory environment and assess and communicate impact of changes on business and product development programs. This position will report to the Vice President of Regulatory Affairs, Gene Therapy. 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Facilitates interpretation of global regulation. * Proactively manages critical issues, taking leadership for the regulatory contribution. * Supports the development of department policies, processes and SOPs. * Provides regulatory due diligence assessments of new business opportunities as required Requirements: * BA/BS Degree required in a health/life sciences or related field. Advanced degree preferred. * A minimum of 13 years in the biotechnology or pharmaceutical industry with at least 8 years of leadership experience managing a regulatory team. * Extensive experience in Regulatory including IND/CTA, NDA/BLA/MAA, lifecycle management, interactions with Health Authorities, and developing and implementing complex regulatory strategies * Understanding of advanced therapy products development * Practical understanding and interpretation of applicable ICH, FDA and EMA guidelines and regulations. * Strong strategic skills including demonstrated ability to make complex decisions and willingness to defend difficult positions. * Strong business acumen and demonstrated ability to make sound decisions that contribute positively to the business. * Excellent operational skills including demonstrated ability in planning, organizing and ability to motivate and lead others. * Excellent verbal and written skills; able to analyze, define and effectively convey difficult and complex issues in a way that accurately and persuasively communicates the issues to internal and external stakeholders, including Senior Management. * Strong negotiating skills and ability to think creatively and develop creative solutions. * Strong personal and interpersonal skills, including the ability to relate to and negotiate with others while maintaining composure and acting with integrity and credibility to build trust * Ability to deal effectively with a variety of personnel across a matrix organization * Strong sensitivity for a multicultural/multinational environment * Ability to prioritize and handle multiple projects simultaneously. * Sense of urgency and perseverance to achieve results * Rare disease and/or gene therapy experience is desirable. * Business travel to be ~10-20% as required. #LI-CZ1 Ultragenyx Pharmaceutical is an equal opportunity employer & prohibits unlawful discrimination based on race, color, religion, gender, sexual orientation, gender identity/expression, national origin/ancestry, age, disability, marital and veteran status. Reasonable accommodation will be provided for qualified individuals with disabilities and for qualified disabled veterans in job application procedures, as required by applicable law. If you have any difficulty using our online application system and need an accommodation due to a disability, you may contact Talent Acquisition by calling: (415) 483-8800 or by emailing us at talentacquisition@ultragenyx.com. For electronic email and mail inquiries, please include a description of your requested accommodation, your name and contact information. See our CCPA Employee and Applicant Privacy Notice. 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. Inquiries on developing a recruiting relationship with us, may be directed to: talentacquisition@ultragenyx.com. Ultragenyx Pharmaceuticals Cambridge MA

Director, Regulatory CMC - Gene Therapy

Ultragenyx Pharmaceuticals