Liver Pre-Clinical Research Lead

Philadelphia, Pennsylvania
Salary not provided
October 26 2021
Organization Type

Primary Duties

The Liver Pre-Clinical Research Lead will manage one or more liver-directed therapeutic programs within the Liver Research group within. 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 and vision within in a group of scientists/research associates.
  • Managing and enabling a high-functioning research team; providing team with training, coaching, scientific advice, and development opportunities, giving timely and constructive feedback on goals and expectations, independently invests in developing management techniques that support an engaged and proactive team environment.
  • Supporting a culture of innovation, change, and constant improvement within Liver R&D
  • Establishing, managing, and optimizing project timelines; ensuring work is completed according to schedule and developing strategies to improve efficiency and ensure quality.
  • Writing and reviewing documents to support regulatory filings, manuscripts, and present results at appropriate scientific meetings.
  • Initiation and active oversight of joint programs with external collaborators; guiding collaborations to productive and meaningful contributions to the field and Spark programs.

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 and cellular approaches as well as in vivo models for hepatic gene therapy programs.
  • Developing strategies to support liver-specific delivery and transgene expression of our gene therapies in laboratory animals.
  • Independently designing and delivering rigorous, scientifically sound IND enabling studies and clearly communicating findings via study report and internal presentations to facilitate product development (e.g. proof-of-concept, dose-ranging, GLP-Tox, biodistribution/vector shedding, etc.)


% of Time Job Function and Description

45% Lead a team of scientists to develop and support liver-targeted gene therapy programs from 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% Guide efforts to identify and validate new target indications. Guide team scientists and research associates with experimental design and data interpretation. Prepare and deliver scientific presentations at internal and external meetings

15%Contribute to study reports and submissions to regulatory agencies

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

5% Hands-on execution of experiments on the bench

Education and Experience Requirements

  • Ph.D. in a relevant scientific discipline (e.g. cell and molecular biology, biochemistry, etc.)
  • Minimum of 5 years of related experience
  • Familiarity with IND enabling studies, regulatory filings, and/or GLP studies is is a plus.
  • Experience in AAV gene therapy and/or in key therapeutic areas, such as hematologic and/or metabolic disorders is a plus
  • Previous experience supervisoring scientists and research associates strongly preferred.
Strong publication record is a plus.

  • Experience managing cross-functional and external collaborations is a plus.

Key Skills, Abilities, and Competencies

  • Hands-on research experience in translational drug development and/or cell and gene therapy
  • Strong motivational and leadership skills capable of building and leading a productive team
  • Strong organizational and time-management skills.
  • Adaptable problem solver and proactive learner
  • Ability to clearly communicate scientific results and strategy to a diverse audience through both written reports and oral presentations
  • Strong scientific knowledgebase and ability to creatively overcome scientific and experimental barriers.


Nearest Major Market: Philadelphia

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.

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