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Pre-clinical Research Lead

Spark Therapeutics
Philadelphia, Pennsylvania. Hybrid and remote options available for select opportunities.
Salary not provided
Closing date
May 27, 2022

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Health Sciences, Clinical Research
Job Type
Research Scientist
Organization Type
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Job Details

Primary Duties:

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

  • Managing and overseeing research activities to support candidate development across the pipeline, including exploratory, proof-of-concept, and IND enabling studies for 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 Pre-Clinical Research Lead position supports translational research into liver-directed gene therapies, 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.
  • Supporting development of enabling technologies that enable expanded access to liver-directed gene therapy and vector re-dosing strategies.
  • Independently designing and executing 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.)


  • Lead a team of scientists to develop and support liver-targeted gene therapy programs from preclinical development to clinical stage. Mentor and develop staff.
  • Ensure departmental goals and objectives are met and aligned to the broader organizational goals and objectives.
  • Guide efforts to develop novel modalities to expand the utility of liver-directed gene therapies. Guide team scientists and research associates with experimental design and data interpretation. Prepare and deliver scientific presentations at internal and external meetings
  • Contribute to study reports and submissions to regulatory agencies
  • Prioritize projects, plan workload, manage resource and personnel scheduling
  • 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.)
  • 3+ years of hands-on experience in protein therapeutics, viral immunology, vaccine biology or related discipline
  • Familiarity with immunobiology, including antibody structure and function
  • Familiarity with IND enabling studies, regulatory filings, and/or GLP studies a plus
  • Direct experience with AAV gene transfer or cell and gene therapies a plus
  • Previous experience supervising scientists and research associates a plus
  • Strong publication record a plus
  • Experience managing cross-functional and external collaborations a plus

Key Skills, Abilities, and Competencies:

  • Strong scientific knowledge of protein biology, protein engineering, or biologic drug development
  • Strong motivational and leadership skills capable of building and leading a productive team
  • Ability to clearly communicate scientific results and strategy to a diverse audience through both written reports and oral presentations
  • Strong organizational and time-management skills
  • Adaptable problem solver and proactive learner
  • Ability to creatively overcome scientific and experimental barriers.

Complexity and Problem Solving:

  • Supports program-level decision making by researching, analyzing, compiling, interpreting, and communicating biological or technical data and literature
  • Develops, optimizes, and approves project-level operational planning with little oversight
  • Supports development and optimization of program-level executional planning for discussion and review
  • Provides guidance and oversight of interpretation of biological, analytical, and technical data to scientific team
  • Researches and provides recommendations on biological models, techniques, and technological platforms

Internal and External Contacts:

  • Interfaces with key contacts outside own area of expertise both internally and externally, including (but not limited to) internal strategic and operational contacts, key opinion leaders in the field, subject matter experts in technological platforms, and study directors at third-party contract resource organizations.
  • Coordinates extensively with internal stakeholders across the Research organization
  • Interfaces with and manages interactions with external vendors or service providers
  • Serves as an internal consultant to management on major matters pertaining to research and planning, providing research synopsis, interpretation, and recommendations


Please be aware that Spark mandates COVID-19 vaccination of all employees regardless of work location. Accommodations may be made in accordance with applicable law.

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.


At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.

Founded in March 2013 as a result of the technology and know-how accumulated over two decades at Children’s Hospital of Philadelphia (CHOP), our investigational therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. Greater understanding of the human genome and genetic abnormalities have allowed our scientists to tailor investigational therapies to patients suffering from very specific genetic diseases. This approach holds great promise in developing effective treatments to a host of inherited diseases. Our initial focus is on treating orphan diseases.

Headquartered in dynamic Philadelphia, we are a diverse, experienced team united in our goal to break down barriers for people and families affected by genetic diseases. As our name suggests, our investigational, one-time therapies are designed to spark healthy biology, and deliver potentially life-altering transformation for people and families affected by genetic disease. Spark is a member of the Roche Group.

Find Us
3737 Market Street
Suite 1300
United States
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