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Lead Scientist- CNS

Date: Jul 26, 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 Research 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:


  • 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.
  • Managing (mentoring) other associate or research scientists
  • Presenting at various leadership meetings as well as conferences per Spark’s recommendations


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


  • In-depth knowledge of neurodegenerative disease/disorder.
  • Experience with delivery techniques targeting the central nervous system such as intracranial injections and have substantial experience with studies in laboratory animals.
  • 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 5 years of hands-on research experience with gene therapies targeting the CNS


Key Skills, Abilities, and Competencies


  • Extensive hands-on experience in CNS/neurodegenerative disease models and manipulations, such as intracerebroventricular and intraparenchymal injections, with experience in CNS gene therapy preferred
  • Experience in general and advanced lab techniques such as molecular cloning, plasmid design and construction, qPCR, ELISA, luciferase and 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 (oral and written) skills
  • 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.
  • Experience with AAV vectors is preferred.



Nearest Major Market: Philadelphia