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Vector Optimization Research Scientist

Spark Therapeutics
Philadelphia, Pennsylvania
Salary not provided
Closing date
Dec 20, 2021

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

Primary Duties

The Research Scientist is a key member of the Discovery Research Team and participates in the development of Spark’s gene delivery platforms. She/he will actively participate in the optimizing gene expression cassettes, facilitating translation from in vitro through in vivo proof of concept.

She/he will be responsible for:

  • Designing, screening, and validating promoters or enhancers for both constitutive and cell-type specific gene expression
  • Conduct unbiased assays such as ATAC-seq, RNA-seq, or ChIP-seq to investigate the mechanisms of gene regulation at both endogenous loci and in the context of AAV episomes
  • Design and evaluate RNA regulatory elements including heterologous introns, UTRs, toehold switches, miRNA binding sites, and RNA aptamers.
  • Collaborating with other Translational Research Scientists to design gene expression cassettes that ensure appropriate functionality of the transgene, including splicing, protein targeting or post-translational processing.
  • Initiating and overseeing joint programs and collaborations with external parties for the development of novel regulatory elements and/or capsids.
  • Writing study reports, manuscripts and present results at appropriate scientific meetings.

The Research Scientist position requires proficiency in molecular biology, biochemistry and gene expression techniques:

  • Multi-fragment cloning into AAV expression plasmids.
  • Practical experience with characterizing epigenetic or chromatin states using NGS-based approaches.
  • Experience with design, execution and data analysis related to experimental studies utilizing both in vitro and in vivo models.
  • Familiarity with gene therapy and/or AAV is preferred.


% of Time Job Function and Description

80% Design, characterize and validate novel gene expression cassettes. This includes executing experiments on the bench, extensive literature review and championing product candidate development.

20% Design experiments, organize, discuss, and record experimental data.

Education and Experience Requirements

  • Typically has a PhD in a biological discipline. Candidate may also have a bachelor’s degree or master’s degree with a minimum of 3 years of applied research experience, exclusive of their degree program.
  • Hands-on research experience with high throughput gene expression analyses (e.g. transcriptional, translational, post-translational, etc.).
  • Familiarity with designing genetic control elements using a combination of molecular, cellular and in vitro/in vivo techniques.
  • Experience with studies in laboratory animal models is preferred.
  • Experience with AAV vectors or non-viral gene therapy is preferred but not required.

Key Skills, Abilities, and Competencies

  • Experience in general and advanced lab techniques such as plasmid design and construction, molecular cloning, qPCR, cell culture, ELISA, plate-based luciferase and/or enzymatic activity assays, SDS-PAGE/western blotting, etc.
  • 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.

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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