Reports to: Associate Director, Gene Therapy Research
Position Summary
Working within Fractyl’s Gene Therapy Research Team, the Sci II / Senior Scientist will be integral to the discovery and development of next generation AAV-based gene therapies for the treatment and cure of diabetes, obesity, and other metabolic diseases. Successful candidates will be responsible for contributing to Fractyl’s lead and pipeline programs with a primary focus on designing, screening, and characterizing genetic constructs targeting metabolic diseases. Achieving this goal will also require developing and optimizing necessary disease models and bioanalytical assays. With a desire to overcome obstacles, the Sci II / Senior Scientist will drive programs forward and ensure timely and effective accomplishment of program goals in close collaboration with the Gene Therapy Research team and external CROs and collaborators.
Primary Responsibilities
- Contribute to the characterization of Fractyl’s lead gene therapy program as it advances to the clinic.
- Pioneer new programs in Fractyl’s gene therapy pipeline from Discovery to Development Candidate nomination.
- Design and optimize genetic constructs to maximize therapeutic effect, including regulatory elements (cell/tissue-specific expression), transgene features (activity), and AAV serotype selection (tropism).
- Perform in vitro screening to assess specificity and efficacy of candidate genetic constructs.
- Develop, optimize, and conduct molecular assays to assess specificity, activity and safety.
- Support generation of biodistribution and transgene expression data and/or other bioanalysis of samples from ex vivo and in vivo studies, as needed.
- Prepare technical reports, summaries, protocols, and quantitative analysis of experimental results.
- Maintain a highly organized and comprehensive electronic lab notebook documenting all work (ELN).
- Identify and bring to Fractyl new technologies, vector concepts, scientific principles, and theories to support our gene therapy mission.
- Mentor, train, and develop junior research scientists.
- Perform all activities in compliance with applicable regulations, Fractyl’s policies and guidelines, including, but not limited to, timely documentation of activities and maintaining all required applicable training.
- Bring a “can do” spirit to work and deliver on other responsibilities as assigned.
Education or Certification Requirements
- PhD in Biology, Biomedical engineering, or other relevant research fields.
Professional Work Experience
- 3-5+ years of highly relevant post-PhD experience either in an academic or industry environment.
- Applicable experience in biotech, pharmaceutical and/or medical device industries preferred.
- Prior vocational experience in related fields a plus (i.e. Co-Ops, Internships, Fellowships, etc.).
- Management and/or mentorship experience of associate scientists and/or scientists preferred.
Qualifications and Skills
- Extensive experience with mammalian cell culture, DNA transfection, and viral transduction (strong preference for experience with AAV).
- Expertise in DNA construct design producing effective gene therapy expression cassettes, including the use of required software (e.g., Benchling, Snapgene, etc.) and the ability to devise cloning/assembly strategies.
- Experience in the design, optimization, and execution of molecular assays quantitating, e.g., gene expression levels, viral titer, protein activity, etc.
- Experience with a variety of molecular and cellular biology techniques, such as molecular cloning, DNA/RNA/Protein purification, PCR including ddPCR and qPCR, flow cytometry, Western blotting, ELISAs, bioassays, plate-based luminescence/colorimetric assays etc., with the ability to learn, troubleshoot, and execute for the goals of the team.
- (Preferred) Past experience in metabolic disease, e.g., T2D, T1D, obesity, etc.
- Experience and/or willingness to work with small and large animal models and tissues as needed to drive forward program goals.
- Contributions to programs that have progressed from discovery research to IND/CTA is highly desirable.
- Track record of challenging oneself to enhance scientific and professional abilities.
Other Essentials and Key Success Factors
- Evidence of "hands-on" experience and expertise.
- Ability to lead fast-paced projects with a keen sense of urgency to get the job done well.
- Highly organized and detail-oriented with a passion to deliver quality results.
- Self-motivated and the ability to work independently while working towards common team goals.
- Proven and successful track record as a team-player and collaborator in small working environments.
- Drives high standards for individual, team, and organizational accomplishment.
- Demonstrated record of intellectual curiosity, innovation and creative problem solving with an entrepreneurial spirit.
- Exhibits an exceptional degree of sustained ingenuity, creativity, and resourcefulness.
- Excellent verbal and written communication skills, with experience translating technical concepts into user-friendly documentation.
- Successful track record of working in high-growth and dynamic organizations.
- Highest levels of professionalism, confidence, personal values, and ethical standards.
Travel
- Limited travel may be required for the role (<5%) for conference attendance and facilitating external collaborations.
The description and requirements outlined above are general; additional requirements may apply.
