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Research Scientist- Liver

Date: Jul 22, 2021

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 Translational R&D team designs and engineers breakthrough therapies that harness the power of the liver as a platform for production and systemic distribution of therapeutics. Research Scientists on the Liver R&D Team drive research efforts from early discovery through proof of concept studies to the clinical setting. She/he coordinates cross-functional efforts within our R&D organization to advance gene therapy programs. 


The Research Scientist is a member of the Liver Therapeutic Area within the Translational Research Department. He/she will be responsible for:

•             Developing strategies to optimize potency and efficacy of gene therapy vectors.

•             Creating novel approaches to enhance specificity, regulatability, and durability of gene therapy vectors.

•             Design and support in vivo studies to assess the efficacy and safety of lead candidate therapeutics.

•             Analyze and summarize experimental findings in the form of study reports, peer-reviewed manuscripts, and oral/poster presentations at scientific meetings.

•             Initiate and manage external research collaborations with contract research organizations and academic investigators in a diverse array of scientific disciplines.




Identify and validate new indication targets and optimize gene expression cassettes to support both existing and new programs in the pipeline. This includes executing experiments on the bench, extensive literature review, championing product candidate development from proof of concept to IND submission, etc.



Prepare sample analysis reports, present results to various internal teams, and write parts of regulatory submissions to regulatory agencies



  • A minimum of 3 years research experience in a biologically relevant field such as molecular biology, cell biology, biochemistry, or genetics/genomics. Familiarity with gene and protein engineering, gene therapy, synthetic biology or biotechnology a plus.
  • Degree in biological sciences required. Master’s degree or PhD preferred.
  • Familiarity with target lead identification/optimization using a combination of molecular, cellular and in vitro/in vivo techniques.
  • Experience with studies in laboratory animal models.
  • Experience with AAV vectors and hepatic gene therapy is preferred, but not required.



  • Highly adaptable and comfortable in an environment with dynamic priority demands.
  • Self-motivated and effective at managing multiple projects in parallel.
  • Strong communication skills, exhibiting the ability to clearly and concisely describe research to a cross-disciplinary audience.
  • Rigorous and conscientious, exhibiting care and thoroughness in data interpretation.
  • Continuous learner, consistently developing and improving scientific understanding and methodology.
  • Proactive and collaborative mindset, exhibiting an enthusiastic team attitude.
  • Meticulous record keeping, documentation, and reporting of research.
  • Independently learn, understand, and utilize current and developing industry practices, techniques, and standards in a rapidly changing field.




Nearest Major Market: Philadelphia