Senior Director, Cmc-qc Small Molecule & Oligonucleotides

Position Summary:

ultraimpact – Make a difference for those who need it most

Ultragenyx is looking for an experienced professional as Senior Director, CMC-QC Small Molecule & Oligonucleotides with a passion and creativity to manage the quality control aspects of product life cycles for rare and ultra-rare diseases.  This role will report the head of Executive Director, CMC-QC.  The key responsibilities will include:

• The Senior Director of Quality Control will provide managerial, technical, CMC, operational and strategic leadership in the development and implementation of all quality control and related activities.
• This will include team leadership, Quality control business process build out, establishment of technical operations, methods transfer, feasibility assessment, qualification/validation/verification and execution of multiple methods.
• The ideal candidate will oversee specifications setting activities, support phase appropriate critical quality attribute determination for all small molecule, ASO, and related assets, lead stability programs at external contract laboratories.
• The Senior Director will be a member of the QC Senior Leadership team, whereby contributing towards harmonized external QC operations for a broad spectrum of therapeutic assets including Small Molecule and Oligonucleotide based therapeutic entities.
• This role will require full collaboration with Analytical Development, Manufacturing Operations, Quality Assurance, and CMC Regulatory Affairs teams.
• Fostering a quality and continuous improvement mindset to ensure that all QC deliverables are cGMP compliant in a phase appropriate fashion to support all clinical phase and commercial programs.
• Managing contract organizations including CMOs and Contract Laboratories (CLs) to ensure appropriate compliance with ICH and regulatory quality standards
• Develop internal system to manage QC technical knowledge base for all Small Molecules & Oligonucleotides assets

The successful candidate must be proficient in QC and Analytical platforms for Small Molecules & Oligonucleotides.  The ideal candidate will have a track record of cGMP and CMC-QC experience.  The candidate has an end-to-end CMC regulatory drug approval process knowledge for Small Molecules & Oligonucleotides, be a self-starter with the ability to work independently under minimal supervision, and who is seeking to be part of an innovative and fast-paced team.  This is a rewarding opportunity to join a commercial Company working on fascinating clinical molecules focused on developing life-transforming therapeutics for patients with rare and ultra-rare diseases.  The individual will be instrumental in bringing the company’s therapeutic products from early-discovery development stage to commercial launch and be a critical part of creating an innovative team for the development of the company’s next generation products.

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:

• Management of Method validations, transfer, testing, life cycle management and investigations for multiple small molecule and oligonucleotide assets. This includes assessment and evaluation for QC fitness of newly transferred methods from analytical developments team as well as contract research organizations.
• QC-CMC activities - Ensure that all CMC stake holders are fully aligned and in harmony to support all development and commercial programs. Support continuous collaborative state with Analytical Development, Technical Operations, Manufacturing, Quality Assurance, Regulatory Affairs, Clinical Development, and Program Management, as well as with external vendors (CROs and CMOs), and Qualified/responsible Persons.
• Close engagement with Analytical Development and Quality Assurance to build a sustainable GMP Quality Control platform to support commercial and growing portfolio of clinical development programs. This includes ensuring harmonized approach to method development that supports the transition and implementation of methods fit for commercial Quality control support.
• Oversight, selection and management of contract QC laboratories and CMOs for multiple analytical assets. This includes technical due diligence, routine performance and methods/product life cycle management.  Including regular visits to ensure sustained quality control and analytical deliverables.
• Support internal and external GMP manufacture and supply of high-quality products for commercial and clinical trials, including oversight of GMP sample handling, testing, QC stability program, QC release and stability data analyses, and method data trending, OOE/OOT/OOS investigations and support Certificates of Analysis generation.
• Develop phase appropriate quality control capabilities, business processes, and procedures to ensure robust product supply, including establishment and support of appropriate processes for specification setting and holistic product control strategy.
• Promote a company-wide culture of safety, quality, and responsible mindset. This includes having appropriate metrics and community ownership of these key cultural traits
• Provide strategic guidance on all QC related agenda for Small Molecules & Oligonucleotides assets. Collaborate with analytical development or CROs on method selection, development, qualification/validation, technical transfer and troubleshooting.
• Execute technical review of analytical data, data integrity and laboratory documentation, method validation protocols, method validation summary reports, and method development reports

Requirements:

• Bachelor’s or master’s degree in Biochemistry or Chemistry or Pharmaceutical Sciences or a related scientific field; PhD degree is highly desirable
• Experience with a minimum of 10-15 years in a GMP environment (Biopharmaceutical Industry) with at least 8-10 years in a CMC-QC environment supporting release and stability management of clinical and commercial assets
• In-depth technical, CMC and regulatory understanding of Small Molecule, ASO, and related compounds QC and understanding of holistic control strategy for therapeutic entities as well as applicable international regulations and standards in all phases of clinical development and commercial assets. Good understanding and experience of Small Molecule, ASO, related compound manufacturing sciences and processes.
• Excellent knowledge in QC methods, including RP-HPLC, IEX, SEC. Expertise and familiarity with most of the following characterization techniques including LC-MS, MALDI-TOF-MS, CD, FTIR, DSC techniques.
• Thorough knowledge in compendial methods and its application of testing for Critical Quality Attributes for small molecule and Oligonucleotide products (Drug substance and Drug product)
• Manage the qualification/validation of analytical test methods for all Product Quality parameters, specification setting, managing stability programs for clinical and commercial products in accordance with ICH/FDA/EMA/USP/Ph. Eur. Guidelines
• Excellent oral and written communication skills in English and European languages (preferably German and/or Italian), and proven ability to work autonomously and manage effectively in a matrix environment.
• Proficiency in MS Office, Word and Excel - Proficiency in statistical analysis (e.g. JMP) software desired
• Attention to detail, excellent organizational skills and the ability to work on multiple projects with tight deadlines
• Excellent interpersonal and verbal communications skills and the ability to deal effectively with a variety of personnel both internally and outside the company
• Travelling to the US head office and other global CMO/CRO sites will be required - 10%