Vector Optimization Lead Scientist

Date: May 31, 2023

Location: Philadelphia, PA, US, 19104

Company: Spark Therapeutics

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 Vector Optimization Lead Scientist will champion the scientific strategy and execution for one or more Vector Optimization programs within the Discovery Research group under Research. They will be responsible for:


  • Engineering novel approaches to enhance specificity, regulatability, and durability of gene therapy vectors
  • Developing or adapting technologies for high-throughput screening of non-coding elements to regulate gene expression cassettes
  • Collaborating with other Therapeutic Area Scientists to contribute to the advancement of pre-clinical programs from early discovery to the clinic.
  • Designing and managing in vivo experiments to assess the performance of optimized regulatory or coding elements
  • Leading innovative efforts to on-board new technologies, such as genome editing or programmable epigenetic regulators
  • Initiating and actively overseeing joint programs with external collaborators.
  • Establishing and managing project timelines and ensuring work is completed according to schedule.
  • Mentoring and training Research Scientists and Research Associates on the Discovery Team.
  • Serving as a subject matter expert on a variety of molecular biology methods and genomics techniques and a go-to resource for troubleshooting.
  • Writing and reviewing documents to support regulatory filings, manuscripts, and present results at appropriate scientific meetings.


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


  • In-depth familiarity with the molecular architecture of non-coding regulatory elements such as promoters and enhancers, optimization of transcription factor binding sites, DNA barcoding, and the application of of molecular, cellular and in vitro/in vivo techniques for screening for transcriptional potency and tissue-specificity.
  • 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.
  • Bioinformatic data mining to uncover mechanisms of epigenetic control of gene expression, mRNA processing, or  control of translation. Examples of such methods include RNA-seq, ATAC-seq, ChIP-seq, and Ribo-seq.
  • Track record of performing technology assessments and due diligence efforts in an industry setting is preferred.




% of Time

Job Function and Description


Identify, develop, and employ high-throughput screening methods to enhance gene expression or tissue-specific control of gene expression


New technology development for tunable gene expression or exploring AAV-adjacent technologies (e.g. genome editing)


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


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


Trainings, lab meetings and administration work

Education and Experience Requirements



  • Ph.D. in relevant scientific discipline (e.g. cell, molecular, or synthetic biology, biochemistry, etc.)
  • Typically has between 5-7 years of hands-on experience in the field of gene therapy.
  • Hands-on research experience, ideally with gene therapies in vivo
  • Familiarity with optimization of lead candidates (nucleic acids) using a combination of molecular, cellular and in vitro/in vivo techniques.
  • Experience with AAV vectors is preferred, but not required.

Key Skills, Abilities, and Competencies

  • 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.

Complexity and Problem Solving

  • Expert ability to critically analyze complex problems, develop potential solutions and evaluate impact to Spark.
  • Ability to assume independent ownership of scientific programs, serving as an internal champion.
  • Acts independently to determine methods and procedures on new assignments.
  • Independently plans work activities and schedule to accomplish objectives.
  • Expert skills in synthesizing research findings and effectively communicating findings and recommendations to senior management independently.
  • Errors in judgment or failure to achieve results would result in the expenditure of moderate amounts of company resources.

Internal and External Contacts


•            Serves as a consultant to management on major matters pertaining to research and plans

•            Interfaces with key contacts outside own area of expertise

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.

Spark Therapeutics does not accept unsolicited resumes/candidate profiles from any source other than directly from candidates.   Any unsolicited resume/candidate profile submitted through our website or to personal email accounts of employees of Spark Therapeutics are considered property of Spark Therapeutics and are not subject to payment of agency fees.

Nearest Major Market: Philadelphia