Sr. Scientist, Gene Therapy and Gene Editing

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 Parkinsons disease and Pompe disease. AskBios 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 peoples 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 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 whats right in every situation. Make clear commitments and follow through.

Position Summary

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

Minimum Requirements

• PhD with 5 years experience (may include post-doctoral work) in Molecular Biology Biochemistry Genetics Biomedical Sciences or a related field; OR Masters 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

Preferred Education Experience and Skills

• 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

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 jobrelated 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 or sending us an email at .

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.