TITLE: Scientist, Analytical Development (mRNA-LNP)
COMPANY: Ultragenyx Pharmaceutical Inc.
LOCATION: South San Francisco, CA 94080
DEPARTMENT: CMC AD/QC
WORK TYPE: FULL-TIME
Why Join Us?
To be a hero for our rare disease patients
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of small molecule, monoclonal antibody, mRNA and gene therapy candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
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!
ultrainnovative – Tackle rare and dynamic challenges
Ultragenyx is looking for an experienced Analytical Development Scientist with the passion and creativity for method development and characterization of great medicines for rare and ultra-rare diseases. The Analytical Scientist will be part of the CMC AD/QC group under the Quality Organization. This position will be responsible for developing novel mRNA lipid nanoparticle (LNP) drug substance and drug product analytical methods to support early stage product development, characterization, and testing within the Process Development Lab at the Ultragenyx South San Francisco site. This position will collaborate closely with cross-functional departments to achieve corporate goals and objectives.
The successful candidate should have a strong experience working with nucleic acid therapeutic in LNP formulation. Past industry or academic experience in either mRNA biology or LNP physicochemical characterization is required. The ideal candidate should be a self-starter with the ability to work in the lab under limited supervision. This position will be instrumental in growing the company’s therapeutic portfolio, 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 of products.
Responsibilities including, but not limited to:
- Design and develop novel biochemical or biophysical methods to analyze and characterize the structure and function of mRNA-LNP
- Elucidate the structure and function of mRNA drug substance, such as higher ordered RNA structure and conformation
- Design and develop LNP encapsulation method to analyze and characterize the LNP integrity, stability, and payload encapsulation efficiency
- Design and develop novel LNP disruption and extraction methods to allow effective analysis of the encapsulated nucleic acids payload
- Utilize Sanger sequencing or next generation sequencing (NGS) based platform methods to analyze mRNA drug substance and characterize product variance
- Design and develop sensitive methods to analyze double stranded RNA (dsRNA) impurities and residual DNA impurities in mRNA drug substance
- Provide routine in-house analytical testing support to process development and formulation development groups
- Perform in-house assay pre-qualification/qualification or assist contract lab to conduct assay qualification and validation following appropriate regulatory guidelines
- Perform timely data trending and statistical analysis to evaluate assay performance
- Author and review method SOP, technical reports, and relevant sections of regulatory IND/BLA fillings
- Maintain and oversee AD/QC analytical method life cycle
Technical Requirements & Skills:
- B.S. with 8+ years or M.S. with 5+ years or Ph.D. with 2+ years of industry experience in Biochemistry, Molecular Biology, Analytical Chemistry, or a related discipline
- Candidate must have at least one of the following experience – technical expertise in either mRNA biology, including gel electrophoresis, qPCR, mRNA sequence analysis using traditional or next generation sequencing (NGS) platforms, or LNP physicochemical properties analysis, for example, particle size, charge, aggregation, and polydispersity analysis
- Understand the common approaches of LNP disruption and payload extraction methods and could apply DoE approach to systematically optimize such conditions
- Familiar with RiboGreen, ELISA and Slot Blot methods is highly desired
- Previous experience with biophysical structure characterization methods, such as light scattering (DLS, MALS), Differential scanning calorimetry (DSC), and circular dichroism (CD) is a plus
- Familiar with analytical method development, qualification, and data analysis and statistical analysis software, such as MS Excel, SoftMax, PLA, and JMP
- Attention to detail, excellent organizational skills and the ability to work on multiple projects
- Excellent oral and written presentation skills and proven ability to work autonomously in a matrix environment