CNS Pre-Clinical Research Lead
Job Opportunity at Spark Therapeutics, Inc

Posted on Mar 14

http://www.sparktx.com    215-220-9300

Location: Philadelphia, PA
Job Type: Full Time
Job ID: W4142250

The Neuroscience Lead (or CNS)  will manage the CNS functional group within the Discovery Research group under Translational Research. He/she will be responsible for:

•    Providing leadership within a group of 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.

The CNS Preclinicnal Lead position requires extensive translational experience with CNS gene therapies, including;

•    In-depth knowledge of neurodegenerative disorders.

•    Experience with delivery techniques targeting the central nervous system such as intracranial injecions 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.

•    Some familiarity with AAV, as well as some knowledge of clinical trial design and regulatory strategy, is preferred.


20%    Hands-on execution of 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%    Lead a team of scientists.  Mentor and develop staff.

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


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

•    Strong organizational skills.

•    Excellent writing and verbal communication skills.

•    Previous management experience preferred, but not required.

•    Strong publication record is a plus.

•    Experience with AAV vectors is a plus.

•    Demonstrate independent problem solving skills of technical issues; develop solutions to a variety of moderate to complex problems. Discuss with supervisor and refer to policies/practices for guidance.

•    Must be comfortable in the fast-paced, biotech start-up environment and interact with multi-disciplinary research teams at Spark.

•    Exercise judgment within defined procedures and practices to determine appropriate action.


•    Ph.D. in relevant scientific discipline (e.g. cell and molecular biology, biochemistry, etc.) 

•    Minimum 5-8 years of hands-on experience in the field of gene therapy for neurodegenerative disorders. 

•    Previous management or supervisory experience strongly preferred.


Transforming Patient’s Lives. Spark Therapeutics is a pioneering gene therapy leader transforming the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. We are embarking on our goal to deliver the first approved gene therapy product in the US for genetic blinding conditions and have a product pipeline addressing other unmet rare genetic diseases. We have an entrepreneurial culture and are looking for talented and passionate professionals who are committed and motivated to making a difference in the lives of our patients. If you share our passion for excellence and are looking for a dynamic work environment that fosters creativity, scientific innovation and team collaboration, you may be a great fit for our team.


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