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Senior Research Investigator I Gene Therapy

Cranbury, NJ

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Come see Amicus Therapeutics in person at the
 Career Fair 2018 in Edison, NJ - Register to attend at  ****Summary of Position 
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 Sr. Investigator will manage one or more supporting scientists to carry out the responsibilities of the Gene Therapy Core. The evaluation of endogenous expression and localization of the therapeutic targets will be required to guide viral vector design parameters. The Sr. Investigator will collaborate with each therapeutic target lead to understand the disease target and the program needs. They will design and execute in vivo studies for the efficacy and safety of gene delivery, appropriate to the disease target, with management of both internal and external resources. 
Roles and Responsibilities 
The Sr. Research Investigator I is responsible for: 
- Collaboration with Therapeutic Target Leads to define the parameters for an appropriate Gene Therapy therapeutic, including choice of promoter, optimization of transgene, and capsid 
- Design, construction, purification, titering, and quality analysis of gene delivery vectors using internal and external resources 
- Analysis of normal target expression in disease-relevant cells and tissues 
- Design, oversight, and interpretation of in vitro and in vivo experiments to evaluate the efficacy of gene delivery 
- Collaboration with internal and external resources to conduct required studies 
- Tracking progress of development of multiple Gene Therapy targets 
- Prioritization of workflows within the Gene Therapy Core including developing the objectives for, and monitoring activities of support personnel. 
- Ensuring documentation of all work in an electronic notebook 
- Analysis and presentation of results to larger scientific group and at external conferences
- PhD with 2 - 5 years' experience in a gene therapy core 
- Proven track record with design, production, and use of AAV and lentiviral gene delivery systems in cell and animal models 
- Successful management of a support team 
- Demonstration of expert knowledge of state-of-the-art technologies associated with development of gene therapy 
- 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 Travel 
- Occasional travel to scientific conferences to present work or for training courses 
How to Apply
Apply at  ****The
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