Research Investigator, Gene Therapy

Amicus Therapeutics
Cranbury, New Jersey
Negotiable based on experience
June 07 2018
Organization Type

Research Investigator


Position Summary

This position will be responsible for the design, production, and characterization of viral vectors for the purpose of gene therapy development towards a range of targets.  The evaluation of endogenous expression and localization of the therapeutic targets will be required to guide viral vector design parameters.  Efficacy of gene delivery will be characterized in vivo, with management of both internal and external resources.

Roles and Responsibilities

  • Design, construction, purification, and titering of gene delivery vectors
  • Analysis of normal target expression in cells and tissues
  • Design, oversight, and interpretation of experiments to evaluate the efficacy of gene delivery
  • Collaboration with internal and external resources to conduct studies
  • Documentation of all work in an electronic notebook
  • Analysis and presentation of results to larger scientific group


  • PhD with 0 – 2 years’ experience in gene therapy
  • Proven track record with design, production, and use of AAV and lentiviral gene delivery systems in cell and animal models
  • Experience with characterization of gene and protein expression in cell and animal models
  • Able to function both independently and as part of a team
  • Demonstration of detailed record keeping and data documentation

Experience and Skills

  • Design, construction, packaging, purification, and titering of AAV and lentiviral gene delivery vectors.
  • Analysis of target gene expression by qRT-PCR and/or RNAseq and protein expression by biochemical or histological methods.
  • Design of in vivo efficacy experiments to demonstrate successful gene delivery and function.


  • Occasional travel to scientific conferences to present work or for training courses

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