CNS Platform Scientist

Philadelphia, Pennsylvania
Salary not provided
October 30 2021
Organization Type
Job Type
Staff Scientist
Spark Therapeutics is seeking a motivated, Platform Scientist to work in a dynamic and collaborative environment to help advance Spark’s CNS pipeline. The Research Scientist is a member of the CNS Platform Technologies Team supporting the creation of an optimal platform for delivery of gene therapies to the CNS. S/he will support the generation and characterization of novel gene therapy medicinal products based on engineered and optimized recombinant adeno-associated virus (AAV) vectors or other delivery methods. Specific responsibilities include:

• Establishing a “CNS toolkit” which enables rapid development of gene therapies for neurodegenerative or neurological diseases.

• Overseeing and facilitating programs which aim to develop vectors with enhanced tropism for CNS tissues or cell types or with other properties which enable safer or less invasive delivery of gene therapies to CNS tissues.

• Developing and implementing technologies which enable detection of AAV genomes and/or transgene expression in CNS tissues and at the single-cell level.

• Implementing non-invasive imaging methods for evaluating the biodistribution of gene therapy vectors and transgenes in vivo.

• Supporting CNS programs, facilitating translation from in vitro through in vivo proof-of-concept in support of current and future therapeutic programs.

• Contribute to joint programs and collaborations with external parties.

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

• Writing study reports, manuscripts and presenting results at appropriate scientific meetings.


Continue ongoing and identify and validate new CNS platform discovery research programs. This includes executing experiments on the bench, extensive literature review, championing product candidate development. Devise, evaluate and trouble-shoot novel strategies to address limitations of current CNS gene therapy approaches.

Contribute with management of external collaborations in novel technologies.

Write study reports, manuscripts and present results at appropriate internal and external scientific meetings.

Education and Experience Requirements

• Typically has a PhD in a scientific discipline (neuroscience, virology, cell and molecular biology, genetics, biomedical engineering).

• Hands-on research experience, ideally with gene therapies and minimally invasive delivery methods targeting the CNS.

• Familiarity with target lead identification/optimization using a combination of molecular, cellular, and in vitro/in vivo techniques.

• Experience with studies in laboratory animal models.

• Experience with AAV vectors and CNS gene therapy is preferred.

Key Skills, Abilities, and Competencies

• We are looking for an individual who is passionate about using technology to enhance the power of gene therapies.

• Ability to design and implement scientific plans with no instructions on routine work and minimal instructions on new assignments.

• In-depth expertise and strong laboratory skills with a diverse range of molecular biology and cell biology techniques relevant to the evaluation of CNS gene therapies.

• Self-motivated to learn and develop new techniques, perform multiple tasks simultaneously, maintain accurate records, develop procedures, and comply with company policies.

• Problem solver, able to persist and overcome scientific challenges and obstacles.

• Strong team player who works closely and effectively with colleagues.

• Energetic and eager to manage multiple activities and priorities.

• Strong organizational skills and dedication to quality in all work tasks and deliverables.

• Comfortable with responsibilities in an entrepreneurial, fast–paced, start-up company environment.

• Excellent writing and verbal communication skills.

Complexity and Problem Solving

• Demonstrate problem solving skills of technical issues; develop solutions to a variety of complex problems which require the regular use of ingenuity and innovation; 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 and ensure solutions are consistent with organization objectives.

• Contribute to efforts to develop and/or acquire novel platform components that enable delivery of gene therapies to the CNS.

• Perform work without appreciable oversight.

Please be aware that Spark mandates COVID-19 vaccination of all employees regardless of work location. Accommodations may be made in accordance with applicable law.

Nearest Major Market: Philadelphia

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.

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