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CNS Preclinical Research Lead

Date: Apr 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 CNS Pre-Clinical Research Lead will lead a research group within the CNS Therapeutic Area focused on developing and testing gene therapies which aim to treat neurodegenerative disease. The successful candidate will have deep subject matter knowledge of neurodegenerative diseases, particularly those diseases involving the accumulation of misfolded or aggregated proteins (e.g. Alzheimer’s disease, Parkinson’s disease, Huntington’s disease or amyotrophic lateral sclerosis). The candidate must also have extensive experience in experimental techniques required for the study of neurodegenerative disease and in methods for evaluating therapeutic efficacy of various interventions including gene therapies, mAbs, and ASOs which are currently being developed to treat neurodegenerative diseases.


This individual will have primary responsibility for interrogating and validating therapeutic targets for gene therapy interventions in neurodegenerative disease, and for progressing a research program based on those target(s) from the earliest exploratory studies through GLP toxicology studies. This individual will also support programs as they advance through clinical development and therefore a basic knowledge of the stages of clinical development/regulatory approval process is desirable. The Pre-clinical Research Lead will manage a group of scientists and research associates working on CNS gene therapy programs, and may additionally manage or collaborate with investigators outside the organization to advance research priorities or to establish new technologies within the team. Familiarity with gene therapy technologies (especially AAV vectors) and delivery methods is advantageous but not required. He or she will be responsible for:


  • Providing leadership within a group of 5-10 scientists/research associates, to develop pre-clinical programs from early discovery to the clinic.
  • Delivering rigorous, interpretable and scientifically sound IND enabling studies to enable product development (e.g. proof-of-concept, dose-ranging, GLP-Tox, biodistribution/vector shedding, etc.)
  • Initiate and oversee joint programs with academic or industry collaborators.
  • Establishing project timelines and ensuring work is completed according to schedule.
  • Training and developing staff at varying levels of experience.




% of Time

Job Function and Description


Lead a team of scientists to develop CNS-targeted programs through preclinical development and to clinical stage.  Mentor and develop staff.

Ensure departmental goals and objectives are met and aligned to the broader organizational goals and objectives.




Identify and validate new indication targets. Design and trouble-shoot experiments based on personal experience and literature review. Prepare and deliver scientific presentations at internal and external meetings



Preparing sample analysis reports and write parts of regulatory submissions to FDA






Provides leadership to team members including managing performance, developing people, hiring and integrating new team members and fostering Spark’s culture, mission, vision and values. 


Education and Experience Requirements


  • Ph.D. in relevant scientific discipline (neuroscience, neurobiology) with minimum 5 years post-PhD hands-on experience (including postdoctoral training) in neuroscience or neurodegeneration research
  • Generally has 5+ years of hands-on research experience in neurodegenerative disorders.
  • Management and/or supervisory experience is a plus


Key Skills, Abilities, and Competencies


  • An energetic and creative person who enjoys working collaboratively and leading a multidisciplinary or cross-functional team and who is able to “multi-task” and adjust rapidly to experimental setbacks or shifting priorities
  • Someone who enjoys and excels at working with others inside and outside the organization to accomplish research goals. Able to initiate and manage external collaborations.
  • Extensive hands-on experience in neurodegenerative disease research including and disease models, culture of iPSC and/or primary culture neurons and glia, biochemical analysis of protein burden, behavioral assays, evaluation of evaluation of neurodegenerative progression and assessment of neuronal network connectivity and plasticity.
  • In-depth understanding of neuroanatomy, CNS cell biology, neurodegenerative processes related to disease or injury
  • Basic knowledge of clinical development stages and regulatory process sufficient to deliver a clinical asset ready for first in human testing
  • Strong organizational skills.
  • Excellent writing and verbal communication skills.
  • Demonstrated ability to hire & integrate new talent into the organization, manage performance, develop people, and model the company’s values & culture
  • Industry experience is a plus


Nearest Major Market: Philadelphia