What are the responsibilities and job description for the Sr. Scientist, Gene Therapy and Gene Editing position at AskBio Inc.?
AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive array of capsids and promoters. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing.
Our vision: Pioneering science to create transformative molecular medicines.
Our mission: Lead innovative science and drive clinical outcomes to transform people's lives.
Our Principles
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.
Job Responsibilities
Agencies: Please do not contact any employee at AskBio about this requisition. Any resume submitted by a recruitment agency to any employee at AskBio, through any medium, will be deemed the sole property of AskBio unless the agency was engaged by AskBio Talent Acquisition team to recruit for that position. All agencies must have a prior executed service agreement with AskBio prior to any search engagement. If a candidate who was submitted outside of the AskBio agency process is hired by AskBio, no fee or payment of any kind will be paid to the agency.
Our vision: Pioneering science to create transformative molecular medicines.
Our mission: Lead innovative science and drive clinical outcomes to transform people's lives.
Our Principles
- Advance innovative science by pushing boundaries.
- Bring transformative therapeutics to patients in need.
- Provide an environment for employees to reach their fullest potential.
- Be a Pioneer. We are not afraid of the impossible and to innovate to make gene therapies accessible to those in need.
- Cultivate Collaboration. Strive to be the best teammate, actively listen, openly communicate, and embrace diverse points of view.
- Embrace Responsibility. We are humbled by the enormity of our mission. We hold a relentless commitment to advance science and clinical outcomes for our patients, families, and caregivers.
- Raise the Bar. Continuously drive improvements and efficiencies. Seek and provide constructive feedback. Have a bias for learning and action.
- Act with Uncompromising Integrity. Be honest, transparent, and committed to doing what’s right in every situation. Make clear commitments and follow through.
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.
Job 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
- 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
- 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
Agencies: Please do not contact any employee at AskBio about this requisition. Any resume submitted by a recruitment agency to any employee at AskBio, through any medium, will be deemed the sole property of AskBio unless the agency was engaged by AskBio Talent Acquisition team to recruit for that position. All agencies must have a prior executed service agreement with AskBio prior to any search engagement. If a candidate who was submitted outside of the AskBio agency process is hired by AskBio, no fee or payment of any kind will be paid to the agency.