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CNS AAV Platform Senior Scientist

Date: Jun 11, 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.

Primary Duties

The CNS AAV Platform Scientist is a key member of the CNS Discovery Research Group and participates in the development of Spark’s CNS-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:


  • Managing in vivo studies employing CNS transgene delivery using AAVs, and tracking the fate of AAV-associated genomes, transcripts, and proteins.
  • Designing high throughput large animal screens and evaluating and interpreting AAV capsid and transgene distribution and expression pattern in the CNS. 
  • 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 CNS-directed candidates.
  • Initiating and overseeing joint programs with external collaborators.
  • Writing study reports, manuscripts and present results at appropriate scientific meetings.


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


  • In-depth knowledge of delivery techniques targeting the CNS and extensive experience leading large animal in vivo studies.
  • Experience designing and managing preclinical in vivo studies.
  • In-depth knowledge of AAV biology. 
  • Practical experience with designing, evaluating, and interpreting studies using high throughput approaches. 
  • Familiarity with target validation and lead identification/optimization, using a combination of molecular, cellular, and in vitro/in vivo techniques for CNS gene therapy programs.
  • Design and construction of optimized gene expression cassettes to increase transgene expression within neuronal cells.
  • Experience with design, execution, and data analysis of experimental studies utilizing both cell-based and in vivo models.




% of Time

Job Function and Description


Work in the labs to perform experiments described above in the primary job duties


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


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


Trainings, lab meetings and administration work



Education and Experience Requirements

  • Ph.D. in relevant scientific discipline required (e.g. cell and molecular biology, biochemistry, etc.)
  • Generally has at least 3-5 years of hands-on research experience with AAV gene therapies targeting the CNS



Key Skills, Abilities, and Competencies

  • Extensive experience in executing in vivo studies for the development of CNS-directed AAV gene therapies required.
  • Practical experience with designing, evaluating, and interpreting studies using high throughput approaches. 
  • Experience in general and advanced lab techniques such as molecular cloning, plasmid design and construction, qPCR, qRT-PCR, ELISA, luciferase and enzymatic activity assays, SDS-PAGE/western blotting, etc.
  • Experience in industry preferred.  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 (oral and written) skills.
  • Works with colleagues as a team player.
  • Strong organizational skills.
  • Strong publication record is a plus.
  • Experience managing external collaborations is a plus.
  • Familiar with computer software commonly used in bio/pharma industries.


Nearest Major Market: Philadelphia