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:

• Advance innovative science by pushing boundaries. 

• Bring transformative therapeutics to patients in need.  

• Provide an environment for employees to reach their fullest potential.  

Our values:

• 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.

Position Summary

The Senior Scientist, Molecular Biodistribution leads and executes biodistribution and gene expression assessment strategies.  This role is responsible for designing fit-for-purpose molecular assays, generating high-quality tissue distribution and transgene expression data, and interpreting results to inform candidate selection, dose strategy, safety assessment, and support regulatory submissions.

The role partners closely with Product Development, Translation Medicine, Tech Ops, and Research and Development among other functions.  The successful candidate combines deep hands-on technical expertise with strong scientific judgment in assay development, data analysis and interpretation, and cross-functional communication.

This is an onsite position in RTP, NC and will report to the Director, Product Development Cores.

Job Responsibilities

Biodistribution Strategy and Study Support

• Design and support biodistribution and gene expression assessment plans for genetic medicine programs

• Define fit-for-purpose tissue collection, processing, and analytical strategies for biodistribution, persistence, and transgene expression assessments

• Contribute to dose selection, route-of-administration evaluation, and tissue targeting assessments for AAV and other genetic medicine platforms

• Evaluate data in the context of mechanism of action, tissue tropism, vector performance, and nonclinical efficacy

Assay Development and Execution

• Develop, optimize, and troubleshoot molecular assays to quantify vector genomes, transgene expression, and related biomarkers in tissues and biofluids

• Apply and oversee quantitative PCR-based and related molecular methods, including qPCR, ddPCR, RT-qPCR, and other nucleic acid quantification approaches

• Establish assay controls, standards, acceptance criteria, normalization strategies, and data quality practices appropriate for regulated and non-regulated studies

• Collaborate with internal stakeholders external partners to ensure reliable assay execution, method transfer, and sample analysis

• Drive continuous improvement in assay sensitivity, specificity, reproducibility, and operational scalability

Data Analysis and Interpretation

• Analyze and interpret biodistribution and gene expression datasets to support program decisions in research and development

• Assess vector genome copy number, transgene RNA expression, and relationships across dose, tissue, timepoint, and platform

• Identify technical or biological sources of variability and recommend follow-up experiments or analytical refinements

• Summarize findings in clear scientific narratives for study reports, governance discussions, and regulatory documentation

• Present results and recommendations to cross-functional teams and senior stakeholders in genetic medicine

Minimum Requirements

• PhD in molecular biology, pharmacology, bioanalytical sciences, genetics, biomedical sciences, or a related discipline with 5+ years’ relevant work experience; or Master’s degree in molecular biology, pharmacology, bioanalytical sciences, genetics, biomedical sciences, or a related discipline with 10+ years of relevant work experience

• 5+ years of relevant experience in biotech, pharma, CRO, or academic translational research settings supporting genetic medicine or related modalities

• Direct experience designing, executing, and interpreting biodistribution and gene expression studies for gene therapy, especially AAV-based programs

• Strong hands-on experience with nucleic acid extraction from tissues and biofluids and with quantitative molecular assay platforms such as ddPCR, qPCR, and RT-qPCR

• Experience supporting preclinical drug development, including non-GLP and/or GLP studies, assay qualification, and regulated documentation

• Demonstrated understanding of nonclinical development considerations for genetic medicines, including tissue tropism, vector persistence, transgene expression, and safety assessment

Preferred Education, Experience and Skills

• Experience with AAV gene therapy development across multiple serotypes, capsids, promoters, or routes of administration

• Experience contributing to regulatory submissions for gene therapy or other advanced therapeutic modalities

• Knowledge of statistical and data visualization approaches used for complex multidimensional nonclinical datasets

AskBio Inc. (AskBio) is an Equal Opportunity Employer and does not discriminate against any employee or applicant for employment because of race, color, religion, gender, sexual orientation, gender identity, national origin, age, disability, veteran status or any other protected status prohibited under Federal, State or local laws. All employment decisions are based on valid job‐related requirements. If you are a qualified individual with a disability or a disabled veteran and are unable or limited in your ability to use or access our website, you may request a reasonable accommodation to express interest in a specific opening by calling us at (919) 561-6210 or sending us an email at [email protected].

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.