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CNS Pre-Clinical Research Lead
Spark Therapeutics, Inc
Job Location: Philadelphia, PA
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 Patients 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.


How To Apply:
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