Sr. Scientist, Gene Therapy and Gene Editing
About The Position
The Sr. Scientist, Gene Therapy and Gene Editing, will serve as a project leader for critical gene therapy and gene editing research initiatives, guiding scientific direction and experimental strategies. This role requires expertise in recombinant AAV biology, gene therapy and gene editing technologies, and drug discovery, with the ability to manage several independent projects covering a broad spectrum of R&D objectives. The Senior Scientist operates as a subject matter expert and is expected to develop innovative approaches for gene therapy/editing, validating and implementing techniques to advance R&D programs. This position is based in RTP (North Carolina) and reports to the Director, Cell Biology.
Requirements
- PhD with 5+ years’ experience (may include post-doctoral work) in Molecular Biology, Biochemistry, Genetics, Biomedical Sciences, or a related field; OR Master’s degree with 10+ years’ industry or related experience in a relevant discipline
- Demonstrated experience in drug development and translating research from discovery through preclinical stages
- Expertise in expression vector design, molecular cloning techniques, and gene therapy cassette design and optimization
- Hands-on experience with quantitative PCR, RT-PCR, and droplet digital PCR (ddPCR)
- Experience with next-generation sequencing (NGS) library preparation and data analysis
- Proficiency in miRNA, shRNA, and siRNA design and application
- Hands-on experience with mammalian cell culture, including primary cells and transduction assays
- Proficiency in protein analysis techniques: SDS-PAGE, Western blot, and immunofluorescence microscopy
- Demonstrated ability to lead cross-functional teams and manage multiple concurrent projects
Nice To Haves
- Experience with emerging gene editing technologies (e.g., CRISPR epigenomic editing, PRINT, or prime editing)
- Track record of publications and/or conference presentations in gene editing or gene therapy
- Experience with in vivo gene therapy studies and translational research
- Familiarity with regulatory requirements for gene therapy IND-enabling programs
- Experience managing external collaborations with academic or industry partners
- People leadership experience, including direct reports and performance management
Responsibilities
- Lead a team of scientists and research associates to design, plan, and execute to advance preclinical gene therapy programs
- Provide scientific leadership and project oversight across multiple gene therapy/editing projects, ensuring timely delivery of high-quality data aligned with R&D objectives
- Design and optimize gene editing cassettes, and gene expression vectors
- Design and optimize AAV-based and non-viral delivery constructs for therapeutic applications
- Develop innovative approaches for gene therapy, validating and implementing new gene therapy and gene editing techniques to advance the field and internal R&D programs
- Oversee experimental design and data interpretation for molecular biology assays including quantitative PCR, RT-PCR, ddPCR, next-generation sequencing, and protein analysis (Western blot, SDS-PAGE, immunofluorescence microscopy)
- Manage project timelines, resources, and internal and external collaborations; develop project strategies and troubleshoot experimental bottlenecks
- Author and review study reports, study protocols, SOPs, regulatory filings, and patent applications
- Present scientific findings at internal meetings and external scientific conferences, representing AskBio in collaborations
- Mentor and train scientists at earlier career stages, promoting scientific growth and development within the team
- Stay current with the latest advancements in gene editing, gene therapy, and AAV biology, driving innovation within the group
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What This Job Offers
Job Type
Full-time
Career Level
Senior
