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Liver Research Lead

Spark Therapeutics
Philadelphia, Pennsylvania
Salary not provided
Closing date
Feb 23, 2022

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Health Sciences, Drug Development
Job Type
Research Scientist
Organization Type
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Job Details

The Liver Research Lead will play a key role in the cross-functional management of one or more liver-directed therapeutic programs within the Liver Research group. S/he will be responsible for:

• Strategic leadership of candidate development from early discovery and proof-of-concept through IND enabling studies for specific liver-targeted programs, providing program objectives to in a group of scientists/research associates.

• Leading cross-functional program development teams (e.g. candidate development teams, asset team sub-teams, etc.)

• Initiating and leading joint programs, partnerships and SRAs with external collaborators.

• Establishing and managing program timelines and budget, resource planning and communicating and creating a path to meet program goals and objectives.

• Training and developing staff at varying levels of experience.

• Planning, authoring and reviewing documents to support regulatory filings, manuscripts, and present results at appropriate scientific meetings.

• Contributing to and operationalizing the strategy around creating a high performing team through enhancing efficiency, organizational structure and education initiatives internally and cross-functionally.

• Participating and acting as an SME in due diligence and business development activities when required by the business.

The Liver 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.)


Lead scientific and cross-functional teams to develop and support liver-targeted gene therapy programs from preclinical development to the 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. Define program strategy and ensure proper design and execution of IND enabling packages. Prepare and deliver scientific presentations at internal and external meetings.

Review study reports and author/review non-clinical sections of regulatory submissions to various agencies.

Prioritize projects, plan workload, and manage resources effectively to achieve on time and on budget objectives. Hire workforce and build high performing teams.

Participate in due diligence and business development activities as required by the business.

Education and Experience Requirements

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

• Typically has 8+ years of related work experience in biotech or a relevant translational area, cell and gene therapy preferred

• Typically has 2+ years of management experience.

• Experience with IND enabling studies, regulatory filings, and/or GLP studies is strongly preferred.

• Strong publication record is a plus.

• Experience managing external collaborations and sponsored research agreements is a plus.

Key Skills, Abilities, and Competencies

• Hands-on research experience, ideally with cell and gene therapies, RNA/DNA therapeutics, or protein therapeutics

• Experience with the drug development process through IND (POC, dose estimation, biodistribution, GLP-toxicology, regulatory requirements, etc)

• Strong organizational and leadership skills.

• Experience with studies in laboratory animals.

• Excellent communication skills (both oral and written)

• Strong analytical skills and problem-solving capabilities

Complexity and Problem Solving

• Internal consultant with significant Subject Matter Expertise

• Considered expert in field within Research and Development

• Develops solutions to complex problems which require the regular use of ingenuity and innovation

• Ensures solutions are consistent with organization objectives

• Work is performed with minimal oversight

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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