Share this Job

CNS Pre-Clinical Research Lead

Date: Nov 13, 2018

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 CNS Pre-Clinical Research Lead position requires extensive translational experience with gene therapies targeting the CNS. The successful candidate would have in-depth 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. He or she should be familiar with delivery techniques targeting the liver and have substantial experience with studies in laboratory animals. Some familiarity with AAV, as well as some knowledge of clinical trial design and regulatory strategy, is preferred. He or she will manage the CNS Pre-Clinical Research functional group within Translational Research. He or she will be responsible for:

• Providing leadership within a group of 4 to 5 scientists/research associates, to develop pre-clinical programs from early discovery to the clinic.

• Initiate and oversee joint programs with academic collaborators. 

• Establishing project timelines and ensuring work is completed according to schedule.

• Training and developing staff at varying levels of experience.


20%    Execute experiments on the bench
40%    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
20%    Prioritize projects, plan workload, manage resource and personnel scheduling
10%    Preparing sample analysis reports and write parts of regulatory submissions to FDA
10%    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.  


• Extensive hands-on experience in CNS/neurodegenerative gene therapy techniques and disease models, such as intracerebroventricular, intraparenchymal injections,  etc. 

• Strong knowledge of Translational research, e.g. familiarity with large animal studies, vector production, etc.

• 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

• Strong publication record is a plus.

• Experience with AAV vectors is a plus.

• Industry experience is a plus


• Ph.D. in relevant scientific discipline (e.g. cell and molecular biology, biochemistry, etc.) with minimum 6 years post-PhD hands-on experience.

• Generally has 8+ years of hands-on experience in the field of gene therapy for neurodegenerative disorders. 

• Previous management or supervisory experience required

Nearest Major Market: Philadelphia

Find similar jobs: