Sr Principal Scientist
We are a gene therapy company changing lives with every genetic advancement.
Asklepios BioPharmaceutical, Inc. (AskBio) is a leading, clinical-stage gene therapy company founded in 2001 based on the work of adeno-associated virus (AAV) gene therapy visionary and pioneer, Jude Samulski, PhD. Since that time, our commitment to developing life-saving medicines that cure genetic diseases has never wavered.
As the only end-to-end gene therapy company in the industry, we’ve built a foundation of therapeutic programs, AAV manufacturing and a rich portfolio of patented technology. Our therapeutic focus includes neuromuscular, central nervous system, cardiovascular and metabolic diseases with clinical trials underway for Pompe disease, Parkinson’s disease and congestive heart failure. Our gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library.
We became a wholly owned, independently operated subsidiary of Bayer AG in late 2020 as a cornerstone of its newly formed Cell & Gene Therapy Platform. Our global headquarters are in Research Triangle Park, North Carolina, a thriving biotechnology hub, and we maintain additional research and development operations in Scotland and France and gene therapy manufacturing in Spain.
We have an opportunity for a Sr Principal Scientist to join the AskBio team in our Research Triangle Park (Durham), NC location and be part of our continued success!
About the role
We’re recruiting for a Principal Scientist (Scientist 1-3) to help support our growth. The Principal Scientist will provide scientific guidance and leadership in the preclinical safety de-risking of AAV-based gene therapies and work in close collaboration with colleagues in Translational Medicine. The Principal Scientist will lead a group to identify and evaluate candidate predictive biomarkers and develop mode of action tests that will appropriately transition from early- to late-phase development.
This role will work directly with a variety of external contract research organizations (CROs)/vendors, as well as academic collaborators and key opinion leaders to execute and expand translational research efforts.
Responsibilities & accountabilities
- Design and oversee studies to characterize the biological consequences of a given mechanism of action to enable generation of a robust clinical development plan to identify relevant mode of action, target patient population, potential combination strategies and mechanisms of resistance
- Directly supervise and mentor 2-3 scientists supporting multiple discovery programs
- Demonstrated understanding of preclinical and clinical disease biology and the ability to apply that knowledge to drug development; demonstrated understanding of the complexities and recent developments in the relevant therapeutic/technical area; and the ability to apply such knowledge to drug development
- Design/implement gene expression profiling for measuring gene delivery in target and non-target cells related to CNS, muscle and liver targets
- Evaluate diverse approaches to optimize gene editing and gene transfer into primary hepatocytes or other organ specific cells and in vivo, including preclinical animal models of disease
- Manages investment risk in individual projects through early identification of key mechanistic questions, the subsequent use of human genetic data and preclinical pharmacological tools to build confidence in rationale and safety
- Validate and optimize methods for monitoring delivery vehicle performance in vitro to address key scientific questions related to target cell interaction, uptake, trafficking, unpackaging and integration when appropriate
- Ability to work in a fast-paced environment with a high degree of independence to effectively address both strategic and tactical challenges, leveraging solid understanding of cell biology
- Coordinate generation and characterization of the cell line collection and sourcing appropriate primary cells related to specific programs
- Partner with in vivo translational teams to implement fit-for-purpose in vitro strategies and support program-specific activities
- Execute research timelines to meet program and corporate objectives
- Foster a collaborative, creative, and rigorous culture of scientific discovery
- PhD in cell biology, chemical biology or molecular biology with 1-2 years of relevant research experience in either cell biology, molecular biology and bioassay development; or an MS with 6 years of relevant industry experience is required
- Experience with gene therapy and gene delivery is preferred
- Experience with AAV molecular biology and expression analysis is preferred
- Familiarity with gene editing methodologies
- Familiarity with generation and interpretation of high-dimensional datasets (single cell RNA-seq, ATAC-seq, Cytof) is preferred
- Working knowledge of protein biochemistry, basic flow cytometry or cell imaging technologies, and next generating sequencing assays is preferred
- Experience with various endpoint assays (qPCR and/or ddPCR, immune-staining (IHC/IFC), immunoassays and Western blot) is preferred
- Ability to work on projects in a highly matrixed environment
- Experience leading cross-functional activities is preferred
- Exceptional critical thinking with analytic skills and trouble-shooting ability
- Must have excellent communication (both written and oral), time management, record keeping and data analysis and collaborative skills
- This role is based in RTP, North Carolina and may require about 10% travel
Live an AskLife™
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