Sr. Scientist Group Leader in vivo 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 have an opportunity for a Sr. Scientist Group Leader in vivo Biology to join the AskBio team!

About the role 

This position is based in RTP and reports to the VP of Product Development. The successful candidate will provide technical and operational leadership and expertise in the development of rAAV gene therapy clinical candidate vectors.

The Group Leader is expected to build and lead an in vivo biology (comparative medicine) team. IVB has responsibility for executing in vivo work related to rAAV vector evaluation and development and characterization of the animal disease models. IVB contributes to the 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 working with rAAV vectors is highly preferred. The Group Leader will be well versed in characterizing in vivo behavior of rAAV vectors in small and large animal models. This includes vector biodistribution studies, dose-ranging studies, functional in vivo readouts including behavioral studies, biomarkers and other efficacy readouts.

The Group Leader 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.

This role will also maintain comprehensive understanding of scientific advances related to rAAV vectors, and transfer appropriate information to the direct manager for business-related or product development purposes. He/she may support due diligence, prepare summaries and presentations, and promote AskBio through presentations at key scientific meetings.

PhD or DVM with a minimum of 8+ years of progressive post-degree experience in gene therapy/vaccines/complex biologics (in the order of preference). Direct experience with characterization of rAAV gene therapy products is highly desired. In-depth biostatistics. Demonstrated experience building and leading high-performing teams. Willingness and ability to work in a fast-paced environment through the team members, as well as hands on.

Responsibilities and Accountabilities

  • Build, lead and manage in vivo biology team
  • Provide operational, technical and supervisory guidance to the team
  • Plan and supervise day to day activities of in vivo scientists
  • Organize daily operations, for example ordering, monitoring animal welfare, breeding, scheduling procedures, managing resources
  • Provide direct scientific oversight in design and execution of in vivo studies
  • Direct responsibility for all experimental work conducted by the team
  • Working knowledge of biostatistics
  • Ensure high quality, reproducibility and timeliness of results
  • Serve as a study director on in vivo studies, or POC on studies done at CRO, develop study protocols and amendments, ensure compliance
  • Coordinate activities at CROs, site visit and inspections
  • Serve as a representative on the cross-disciplinary teams
  • Manage projects, both internal and collaborations, including timelines and budgets
  • 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
  • Ability to adeptly handle 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