Scientist II AAV Biology

About us 

Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company founded in 2001 based on the work of adeno-associated virus (AAV) gene therapy pioneers, Jude Samulski, PhD, and Xiao Xiao, PhD. We are driven to change lives with every clinical advancement and turn hope into cures with new possibilities for genetic medicine. We currently have clinical trials ongoing for late-onset Pompe disease and congestive heart failure.

AskBio is headquartered in Research Triangle Park, North Carolina, a thriving biotechnology hub, and has additional research and development facilities in Scotland and France and gene therapy manufacturing in Spain.

We are excited for our next Scientist II AAV Biology to join the AskBio team!

About the role 

This position is based in RTP and reports to the Director, Molecular Biology. The role will conduct experimental bench work in support of development of rAAV gene therapy clinical candidate vectors and be a member of the molecular biology team (MBT). MBT has responsibility for cassette design and optimization, cassette screening and delivery of the lead and back-up clinical candidate vectors for various indications. Areas of interest to AskBio include CNS, neuromuscular, liver systemic and liver metabolic rare diseases.

Therapeutic strategies may include gene replacement, gene knock down, gene modulation, and combinations of those approaches. Therapeutic modality is rAAV, thus experience with developing rAAV vectors is required. This Scientist II will have direct experience with various molecular biology techniques, including quantitative PCR and RT PCR (preference for direct experience with ddPCR), expression vector design, NGS, miRNA< shRNA, off target effects, RNAScope, mammalian cell culture including iPS and primary cells, in vivo animal studies, expression of recombinant proteins and functional characterization of the recombinant proteins.

Scientist II will develop and maintain relationships with key internal leaders and organizations, collaborating laboratories, and CROs and will collaborate with the capsid development group in R&D and aid in characterization of the novel AAV capsids.

Scientist II will maintain comprehensive understanding of scientific advances related to rAAV vectors and transfer appropriate information to the direct manager. He/she may support due diligence, prepare summaries and presentations, and promote AskBio through presentations at key scientific meetings.

PhD in molecular biology, biochemistry or other relevant discipline with 2-6 (MS with 5-9, BS with 9-13) years of progressive post degree experience in gene therapy/vaccines/complex biologics product development roles (in the order of preference). Direct experience with development of rAAV gene therapy products. Willingness and ability to work in a fast-paced environment.

Responsibilities and Accountabilities

  • Design, plan and conduct experiments
  • Analyze data and prepare reports
  • Help plan and supervise day to day activities of junior research scientists
  • Provide direct scientific oversight in design and execution of experiments by junior scientists
  • Ensure high quality and reproducibility of results
  • Contribute to delivering clinical candidate vectors for translational programs
  • Manage experiments, including timelines and budgets
  • Ensure lab areas are clean and well organized and equipment, supplies and raw materials are available for use and safety protocols are followed
  • Contribute to/author study reports, study protocols, SOPs and regulatory filings
  • Excellent communication skills and work well in a group work environment
  • Contribute to technical discussions and investigations within the cross-disciplinary program and project teams
  • Detailed and organized

About you 

  • Ability to develop rAAV products regardless of the therapeutic area by collaborating with the subject matter experts in research, translational, clinical and regulatory organizations
  • Experience working with cross-disciplinary teams, as well as being individual contributor
  • Willing to help to more junior members of the team
  • Ability to multi-task among multiple projects and teams and work independently in a fast-paced, highly interactive environment
  • Excellent interpersonal skills with the ability to interact effectively with people, internally and externally, is required
  • Must be self-motivated, organized and capable of working independently as well as in a collaborative/group environment
  • Should possess strong oral and written communication skills
  • Good judgment and innovation to achieve a solution within standard practices and procedures
  • Experience with global teams, especially Spanish- and French-speaking, a plus