CNS Senior Research Scientist at Spark Therapeutics in Philadelphia, PA

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.

The Senior Research 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.

Responsibilities

% of Time

Job Function and Description

70%

Work in the labs to perform experiments described above in the primary job duties

15%

Design experiments, organize, discuss, and record experimental data.

10%

Generate technical reports, prepare presentation slides, generate novel concepts.

5%

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.