Reporting to the Director of the Gene Therapy Program (GTP), the Director of Research – Neurodegenerative oversees all aspects of neurodegenerative disease research at the GTP at PENN. This role will be responsible for establishing processes for mentoring and leading SRIs in research projects. This role directly oversees and manages a team of SRIs and technicians. In consultation with the PI of the Gene Therapy Program, the Director of Research – Neurodegenerative will assess the rare disease programs that are brought into GTP for scientific research. The individual should have in-vivo pharmacology expertise for preclinical translational programs as well as expertise in testing the efficacy of therapeutic interventions for neurodegenerative disorders.
GTP is a complex R&D organization consisting of research laboratories supported by multiple core functions which provide internal capacity to perform gene transfer studies from basic research through phase 1 proof-of-concept human trials. We currently employ 160 FTEs with an annual operating budget of $50M based on a wide array of funding sources. The R&D enterprise has at its core a translational infrastructure that includes: technology discovery, specialized immunology assays operating under GLP, a Vector Core and Manufacturing unit that conducts GMP-based assays and oversees CMOs in vector production, and a large non-clinical program for animal testing under GLP, including nonhuman primate and mouse vivaria; GTP collaborates with the Department of Medicine in the organization and conduct of phase 1 trials. In addition to these translational programs, GTP is involved in an ambitious vector discovery program called AAV 3.0 which is being leveraged against standard gene replacement therapy programs, as well as research in genome editing. These programs involve multiple federal, nonprofit and commercial partners.
The Director of Research will be asked to build a team of SRIs to support GTP’s neurodegenerative research efforts.
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