Principal Scientist, Translational Gene Therapy RNA therapeutics

Pfizer's Rare Disease Research Unit (RDRU) focuses on developing drugs for treating rare diseases. The Gene Therapy Translational Research group is part of the RDRU focused on gene transfer approaches to treat rare genetically defined diseases that focus on the CNS, neuromuscular, and liver (systemic and metabolic) therapeutic areas. The Principal Scientist will be part of a larger interdisciplinary team to advance Pfizer's gene therapy program developing RNA based approaches to treat rare diseases. The role requires knowledge and expertise in RNA drug discovery and rare diseases. The role requires expertise in designing and evaluating RNA therapeutics using high throughput in vitro screens, next generation sequencing methodologies and data analysis, RNA delivery systems, and understanding of immune responses to RNA therapeutics. The Principal Scientist and his/her team will be involved in early RNA drug development, perform cell-based and biochemical assays, and develop in vivo disease models. The individuals are also required to work in collaboration with Biomedicine design, Medicinal Chemistry, and Drug Safety teams to advance innovative drugs to the clinic. The role requires having a deep knowledge of human disease and RNA biology to advance the project from ideation to execution of all preclinical studies.

Role Responsibilities

• Independently develop RNA based disease targets

• Design, perform and investigate in vitro and vivo studies to assess target validation and proof-of-concept studies

• Genome editing (evaluate nucleases, CRISPR/Cas9, guide RNA design, in vitro and in vivo target assessment, NGS data analysis)

• Perform various biochemical, cell biology, molecular biology, and immunology assays

• Partner with colleagues working on Gene Therapy projects within the rare disease and other partner lines

Qualifications

• Ph.D. in the life sciences with 5+ years of relevant experience in the field of RNA therapeutics and lipid nanoparticles

• Prior experience in gene editing technologies and next generation sequencing a must

• Prior experience in rare disease utilizing small and large animal models is desired.

• Evidence of extensive scientific accomplishments in the form of publications and presentations.

• Evidence of experience working in cross-disciplinary teams.

• Exceptional communication and presentation skills and demonstrated ability to work independently as well as part of a team.

Other Job Details

Eligible for Relocation Package

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Research and Development