Research Scientist Liver Research Group
Job Opportunity at Spark Therapeutics, Inc

Posted on Jul 21

http://www.sparktx.com    215-220-9300

Location: Philadelphia, PA
Job Type: Full Time
Job ID: W4162216

The Research Scientist is a member of the Liver Therapeutic Area within the Translational Research Department. S/he will be responsible for:
  • Design and construction of optimized gene expression cassettes to drive therapeutic transgene expression derived from liver cells.
  • Experimental design, execution and data analysis for liver-directed research programs.
  • Write study reports, manuscripts and present results at appropriate scientific meetings.
  • Initiate and oversee joint programs with internal and external collaborators.

  • 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.

  • Experience in general and advanced lab techniques such as molecular cloning and plasmid design and construction, qPCR, ELISA, cell culture, plate-based luciferase and/or enzymatic activity assays, SDS-PAGE/western blotting, etc.
  • Experience with studies in laboratory animals.
  • Familiarity with IND enabling studies, regulatory filings, and GLP studies is a plus.
  • Strong analytical skills and problem-solving capabilities.
  • Proven ability to work independently in hands-on laboratory setting, self-motivated to learn and develop new techniques, perform multiple tasks simultaneously, keep accurate records, follow instructions, and comply with company policies.
  • Excellent communication skills (both oral and written).
  • Familiar with computer software commonly used in bio/pharma industries.
  • Works with colleagues as a team player.
  • Strong organizational skills.
  • Strong publication record is a plus.

  • Typically has a PhD in a scientific discipline and 3 years of related experience. May also have a Bachelor’s Degree or Master’s degree and 3 years of related experience.
  • Hands-on research experience, ideally with gene therapies targeting the liver.
  • 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.


Transforming Patient’s Lives. Spark Therapeutics is a pioneering gene therapy leader transforming the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. We are embarking on our goal to deliver the first approved gene therapy product in the US for genetic blinding conditions and have a product pipeline addressing other unmet rare genetic diseases. We have an entrepreneurial culture and are looking for talented and passionate professionals who are committed and motivated to making a difference in the lives of our patients. If you share our passion for excellence and are looking for a dynamic work environment that fosters creativity, scientific innovation and team collaboration, you may be a great fit for our team.

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