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Translational Science Lead - CNS

Spark Therapeutics
Philadelphia, Pennsylvania. Hybrid and remote options available for select opportunities.
Salary not provided
Closing date
Jul 12, 2022

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Life Sciences, Biotechnology
Organization Type
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Job Details

Primary Duties

The Translational Sciences and Companion Diagnostics team is seeking an experienced scientist to develop and lead the execution of translational strategy for CNS-directed gene therapy programs at Spark.

The Translational Science lead for CNS-directed programs will serve on Compound Development Teams for pre-clinical stage programs and on Non-Clinical Study Teams for clinical stage programs. The overall goal is to advance our understanding of the vector, the platform, anti-vector immune responses, and disease biomarkers, using learnings in phase 1/2 to optimize phase 3 designs and apply knowledge from all clinical trials back to the pre-portfolio and early pipeline. Areas of translational discovery include mechanisms of action, patient enrichment strategies, novel endpoints, and potential new indications. The scope of biomarkers for CNS-directed gene therapy will include molecular, imaging (MRI, PET), and wearable devices. For programs where a clear patient selection strategy is established, the translational science lead will also be responsible for partnering externally to develop companion diagnostic assays in support of clinical development, launch and life cycle management.

Towards these ends, the lead will oversee the development of translational plans for clinical and relevant preclinical research programs. This will include formulating the scientific and clinical rationales, assays, and translational aspects of clinical protocol design and execution. In addition to learning from individual clinical studies, the Translational Science Lead, CNS will spearhead efforts to integrate learning across programs. Operational responsibilities include oversight of data generation, analysis, and data sharing. To achieve these aims, the lead will need to establish strong connections with internal functional groups across the organization, particularly with the Research Therapeutic Areas, Clinical Development teams, Data Sciences, and Biometrics. External collaborations will also be essential to access expertise in disease biology and biomarker technology.


    Manage the translational strategy and implementation for CNS-directed assets. Develop and implement the translation biomarker strategy for the asset, participating in the Compound Development and Non-Clinical Study Teams. This will include achieving consensus on the key scientific questions, the assays and samples needed to address them, and the analytic approach to the resulting data in concert with Research Therapeutic Area leads, Clinical Development leads and biomarker leads in Biometrics. Work with the preclinical research teams to choose suitable and informative model systems (in vitro or in vivo) to answer key questions and to ensure best translatability of results Work with preclinical research teams to acquire key data in human patients/samples which can validate a particular target or approach Coordinate the analysis and sharing of translational results with Non-Clinical Study Teams, Asset Teams, and to wider audiences internally and externally. Interact with internal and external experts and CROs to ensure that Spark can bring state-of-the-art technology and analysis to our translational plans. Contribute to the biomarker sections for clinical protocols, investigator brochures, clinical study reports, licensing applications, and other regulatory documents.

Education and Experience Requirements

    PhD with 12+ years of experience, or MD with 8+ years of experience, in neuroscience, molecular biology, immunology, or related field and 5+ years of relevant experience in CNS translational research and/or clinical trial experience, preferably in an industry setting. Statistical and computational biology experience are a major plus.

Key Skills, Abilities, and Competencies

    Familiarity with neurosurgical procedures associated with administration of CNS-directed gene therapies is highly desirable Experience with clinical immunogenicity of therapeutics and / or gene therapy is a plus Familiarity with techniques frequently used in Spark translational research, such as antibody-based protein detection and quantitation, imaging (eg, MRI, PET) and functional testing. Excellent written and oral presentation skills. Experience working with matrixed teams and project management preferred.

Complexity and Problem Solving

  • Decision-making will be necessary to identify and prioritize scientific questions/hypotheses and the associated assays and analysis plans, balancing them with operational considerations within clinical trials

Internal and External Contacts

CDT and NCST leads

Therapeutic area representatives/partners

Data science representatives/partners


Academic collaborators


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.


At Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challenge the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.

Founded in March 2013 as a result of the technology and know-how accumulated over two decades at Children’s Hospital of Philadelphia (CHOP), our investigational therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. Greater understanding of the human genome and genetic abnormalities have allowed our scientists to tailor investigational therapies to patients suffering from very specific genetic diseases. This approach holds great promise in developing effective treatments to a host of inherited diseases. Our initial focus is on treating orphan diseases.

Headquartered in dynamic Philadelphia, we are a diverse, experienced team united in our goal to break down barriers for people and families affected by genetic diseases. As our name suggests, our investigational, one-time therapies are designed to spark healthy biology, and deliver potentially life-altering transformation for people and families affected by genetic disease. Spark is a member of the Roche Group.

Find Us
3737 Market Street
Suite 1300
United States
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