Liver Pre-Clinical Research Lead Jobs in Philadelphia, PA at Spark Therapeutics

Title: Liver Pre-Clinical Research Lead

Company: Spark Therapeutics

Location: Philadelphia, PA

Salary: $80K — $100K *

Category: Pharmaceuticals & Biotech

Job Description

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 andin vitro/in vivotechniques 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.)



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.


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


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


Prioritize projects, plan workload, manage resource and personnel scheduling


Hands-on execution of experiments on the bench.


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.


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.

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Valid through: 9/13/2020


About Spark Therapeutics