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Liver Pre-Clinical Research Lead

Date: Aug 1, 2019

Location: Philadelphia, PA, US

Company: Spark Therapeutics

Join the Spark Team


We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our shared mission is to challenge the inevitability of genetic disease by discovering, developing, and delivering treatments in ways unimaginable – until now.


We don’t follow footsteps. We create the path.


The Liver Pre-Clinical Team Lead will manage one or more liver-directed therapeutic programs within the Liver Research group under 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 in 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 Team Lead position requires extensive translational experience with gene therapies targeting the liver, including:


• In-depth familiarity with target validation and lead identification/optimization, using a combination of molecular, cellular and in vitro/in vivo techniques for hepatic gene therapy programs. 

• Familiar with delivery techniques targeting the liver and have substantial experience with studies in laboratory animals. 

• Experience in a AAV gene therapy and/or in key therapeutic areas such as Hemophilia and/or Lysosomal Storage Diseases, is preferred. 

• Experience with pre-clinical proof of concept and IND enabling study design and regulatory strategy, is preferred. 


35% 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. 

30% 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% Preparing sample analysis reports and write parts of regulatory submissions to regulatory agencies

10% Prioritize projects, plan workload, manage resource and personnel scheduling

10% Hands-on execution of experiments on the bench


Hands-on research experience, ideally with gene therapies targeting the liver

• Strong organizational skills.

• Experience with studies in laboratory animals.

• Familiarity with IND enabling studies, regulatory filings, and GLP studies is a plus

• Excellent communication skills (both oral and written)

• Previous management experience preferred, but not required.

• Strong analytical skills and problem-solving capabilities

• Strong publication record is a plus.

• Experience with AAV vectors is preferred.

• Experience managing external collaborations is a plus.


• Ph.D. in relevant scientific discipline (e.g. cell and molecular biology, biochemistry, etc.) 

• Typically has between 4-7 years of hands-on experience in the field of gene therapy. 

• Previous management or supervisory experience strongly preferred.

Nearest Major Market: Philadelphia

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