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Sr. Research Scientist- Liver

Date: Aug 14, 2019

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 Research Scientist is a key member of the Liver Research Group and participates in the development of Spark’s liver-targeted gene therapy products. She/he will actively participate in the development of novel targets, facilitating translation from in vitro through in vivo proof of concept.  


He/she will be responsible for:


• Collaborating with other Translational Research Scientists to develop pre-clinical programs from early discovery to the clinic. 

• Designing and conducting in vitro and in vivo experiments to optimize pre-clinical development of liver-directed candidates.

• Initiating and overseeing joint programs with external collaborators. 

• Writing study reports, documents to support regulatory filings, manuscripts and present results at appropriate scientific meetings.


The Senior Research Scientist position requires extensive experience with molecular biology, biochemistry and immunoassays including:


• Design and construction of optimized gene expression cassettes to increase transgene expression derived from liver cells.

• Experience with design, execution, and data analysis of experimental studies utilizing both cell-based and in vivo models.

• Some experience or familiarity with liver-directed gene therapy and/or AAV vectors is preferred.



70% 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.

15% Design experiments, organize, discuss, and record experimental data.

10% Generate technical reports, prepare presentation slides, generate novel concepts.    

5% Trainings, lab meetings and administration work


• 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.

• Experience managing external collaborations is a plus.


• Typically has a PhD in a scientific discipline and 3-5 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

Nearest Major Market: Philadelphia

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