Lead Scientist (Investigator) Liver Team Spark Therapeutics Spark Therapeutics 15 reviews

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.


The Liver Translational R&D team focuses on transformative therapies for patients with rare diseases. We are seeking a Lead Scientist (Investigator) to engineer and execute the development of liver-directed AAV gene therapies. She/he should have working knowledge of the following areas and be a subject matter expert in a least one of these (adeno-associated virus biology, metabolic or hematological diseases, synthetic biology, and/or immunology). Come join our exciting, multi-disciplinary, and talented team at Spark Therapeutics, a recognized leader in the field of gene therapy.

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 managing in vivo experiments to prioritize lead candidate selection, proof-of-concept studies, dose ranging, as well as GLP-TOX studies.
  • Engineering novel approaches to enhance specificity, regulatability, and durability of gene therapy vectors
  • Identifying and validating new indication targets and optimize gene expression cassettes to support both existing and new programs in the pipeline.
  • Mentoring and training Research Scientists and Research Associates on the Liver Team.
  • Serving as a repository of knowledge on a variety of biochemical/analytical techniques and a go-to resource for troubleshooting.
  • Analyzing and summarize experimental findings in the form of study reports, peer-reviewed manuscripts, and oral/poster presentations at scientific meetings.
  • Authoring, reviewing, and/or finalizing study reports, and present results to various internal teams. Contribute to regulatory submissions at regulatory agencies.
  • Initiating and managing external research collaborations with contract research organizations and academic investigators in a diverse array of scientific discipline
  • Independently analyzing datasets and synthesize pre-clinical finding to communicate project milestones to corporate leadership.

Summary continued….

The Lead Scientist position requires extensive experience with molecular biology, biochemistry and immunoassays including:

  • Design and construction of optimized gene expression cassettes to increase transgene expression derived from liver cells.
  • Experience with design, execution, and data analysis of experimental studies utilizing both cell-based and in vivo models.
  • Experience managing in vivo studies employing transgene delivery using AAVs, and tracking the fate of AAV-associated genomes, transcripts and proteins in vivo.



Identify and validate new indication targets and optimize gene expression cassettes to support both existing and new programs in the pipeline. This includes executing experiments on the bench, extensive literature review, championing 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, generate novel concepts


Trainings, lab meetings and administration work


  • Typically has a PhD degree and post-doctoral training in the life-sciences. A minimum of 5 years of additional research experience.
  • Hands-on research experience, ideally with gene therapies targeting the liver
  • Familiarity with target lead identification/optimization using a combination of molecular, cellular and in vitro/in vivo techniques.
  • Experience with IND enabling studies, regulatory filings, and GLP studies is preferred.
  • Experience with AAV vectors and hepatic gene therapy is preferred, but not required.


  • 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, etc.
  • Experience with studies in laboratory animals.
  • Strong analytical skills and problem-solving capabilities
  • Strong track-record of leadership on a multidisciplinary team. Highly collaborative both on team-based and cross-functional research projects.
  • Strong publication/scientific presentation/patent record.
  • Ability to work independently and adaptable to rapidly-evolving priorities.
  • Able to multi-task, keep accurate records, follow instructions, and comply with company policies.
  • Excellent communication skills (both oral and written).
  • Familiar with computer software commonly used in bio/pharma industries.


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