Sr. Research Scientist – Ocular 1

Spark Therapeutics
September 17, 2022
Philadelphia, PA
Job Type


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.

Primary Duties

The Sr. Research Scientist is a key member of the Ocular Research Group and participates in the development of Spark's Ocular gene therapy products. She/he will actively participate in the development of the pre-clinical pipeline, from identification of lead candidates through proof of concept and IND-enabling studies. She/he will lead efforts in vector design and cloning, identification of efficacy readouts in the different animal models as well as assay development. She/he will also be responsible for performing data analysis independently and communicating scientific findings to leadership. In addition, the Senior Scientist serves as a subject matter expert for more junior members of the team and supports research efforts in other areas of the Translational R&D Department.

He/she will be responsible for:

* Developing strategies to optimize potency and efficacy of gene therapy vectors.
* Creating novel approaches to enhance specificity, tolerability and durability of gene therapy vectors.
* Designing and supporting in vitro and in vivo studies to assess the efficacy and safety of lead candidate therapeutics.
* Analyzing and summarizing experimental findings in the form of study reports, peer-reviewed manuscripts, and oral/poster presentations at scientific meetings.
* Managing external research collaborations with contract research organizations and academic investigators in a diverse array of scientific disciplines.
* Mentoring and training Research Scientists and Research Associates on the Ocular Team.

The Senior Research Scientist position requires extensive experience with molecular biology, biochemistry and assay development including:

* Experience with gene therapy and/or AAV vectors
* Design and construction of optimized gene expression cassettes to increase transgene expression derived from different types of retinal cells.
* Experience with design, execution, and data analysis of experimental studies utilizing both cell-based and in vivo models.
* Familiarity with in vivo ocular techniques is a plus.


Validation of new indication targets and optimization of gene expression cassettes to support both existing and new programs in the pipeline. This includes executing experiments on the bench, extensive literature review, and overseeing product candidate development from proof of concept to IND submission, etc.

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

Generate technical reports, prepare presentation slides, present results to various internal teams, and write parts of regulatory submissions to regulatory agencies.

Trainings, lab meetings and administration work

Education and Experience Requirements

* PhD degree in a biologically relevant field such as molecular biology, cell biology, biochemistry, or genetics/genomics required
* At least 3+ years of post-doctoral training or equivalent industry research experience.
* Subject matter expert in the fields of gene and protein engineering, gene therapy, biotechnology or structural biology.
* Experience with AAV vectors and ocular gene therapy
* Familiarity with target lead identification/optimization using a combination of molecular, cellular and in vitro/in vivo techniques.

Key Skills, Abilities, and Competencies

* Strong experience in general and advanced lab techniques such as molecular cloning and plasmid design and construction, qPCR, ELISA, cell culture, plate-based luciferase and/or enzymatic activity assays, SDS-PAGE/western blotting, WES, etc.
* Experience with studies in laboratory animals.
* Proven ability to work independently in hands-on laboratory setting, self-motivated to learn and develop new techniques and perform multiple tasks simultaneously.
* Meticulous record keeping, documentation, and reporting of research.
* Rigorous and conscientious, exhibiting care and thoroughness in data interpretation
* Strong analytical skills and problem-solving capabilities.
* Excellent communication skills (both oral and written), exhibiting the ability to clearly and concisely describe research to a cross-disciplinary audience.
* Ability to mentor junior team members.
* Proactive and collaborative mindset, exhibiting an enthusiastic team attitude.
* Goal driven and strong organizational skills.
* Strong publication record is a plus.
* Familiarity with IND enabling studies, regulatory filings, and GLP studies is a plus
* Experience managing external collaborations is a plus.

Complexity and Problem Solving

* Senior level role with demonstrated experience
* Develops solutions to a variety of complex problems
* May refer to established procedures and policies
* Worked is performed under general direction
* Participates in determining objectives of assignment
* Plan schedules and arranges own activities in accomplishing objectives
* Work is reviewed upon completion for adequacy in meeting objectives
* Complete understanding and application of principles, concepts, practices, standards

Internal and External Contacts


* Represents department as a prime contact on contracts or projects
* Interacts with senior internal/external personnel on significate matters often requiring coordination between organizations/departments/teams
* Interfaces with senior internal/external personnel in own area of expertise

Other Job Requirements

Travel may be required, but infrequently and for short periods of time. Responsible and detail-oriented working practice is needed. Ability to prioritize work stream to meet complex deadlines for multiple simultaneous projects.


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.

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