Liver Pre-Clinical Research Lead

The Liver Pre-Clinical Research Lead will manage one or more liver-directed therapeutic programs within the Liver Research group within Translational Research. S/he will be responsible for:

• Directing and overseeing candidate development from early discovery and proof-of-concept through IND enabling studies for specific liver-targeted programs, providing leadership within a group of scientists/research associates.
• Initiation and active oversight of joint programs with external collaborators.
• Establishing and managing project timelines and ensuring work is completed according to schedule.
• Training and developing staff at varying levels of experience.
• Writing and reviewing documents to support regulatory filings, manuscripts, and present results at appropriate scientific meetings.

The Liver Pre-Clinical Research Lead position supports translational research of gene therapies targeting the liver, including:

• Target validation and lead identification/optimization, by using a combination of molecular, cellular and in vitro/in vivo techniques for hepatic gene therapy programs.
• Developing strategies to support liver-specific delivery and transgene expression of our gene therapies in laboratory animals.  
• Delivering rigorous, interpretable and scientifically sound IND enabling studies to facilitate product development (e.g. proof-of-concept, dose-ranging, GLP-Tox, biodistribution/vector shedding, etc.)

Responsibilities:

45%  

Lead a team of scientists to develop liver-targeted programs through preclinical development and to clinical stage.  Mentor and develop staff.

Ensure departmental goals and objectives are met and aligned to the broader organizational goals and objectives.

25%

Identify and validate new indication targets. Design and trouble-shoot experiments based on personal experience and literature review. Prepare and deliver scientific presentations at internal and external meetings

15%

Prepare sample analysis reports and write parts of regulatory submissions to regulatory agencies.

10%

Prioritize projects, plan workload, manage resource and personnel scheduling

5%

Hands-on execution of experiments on the bench.

Requirements:

• Ph.D. in a relevant scientific discipline (e.g. cell and molecular biology, biochemistry, etc.)
• 5 plus years of hands-on experience in the field of gene therapy or a relevant field of expertise.
• Familiarity with IND enabling studies, regulatory filings, and/or GLP studies is required
• Experience in AAV gene therapy and/or in key therapeutic areas, such as hematologic and/or metabolic disorders is a plus
• Previous management or supervisory experience  preferred.
• Strong publication record is a plus.
• Experience managing external collaborations is a plus.

Competencies:

• Hands-on research experience, ideally with gene therapies targeting the liver.
• Strong organizational skills.
• Experience with studies in laboratory animals.
• Excellent communication skills (both oral and written).
• Strong analytical skills and problem-solving capabilities.