Senior Scientist, Rare Disease

Cambridge, Massachusetts
July 18 2021
Position Type
Full Time
Organization Type
Job Type
Senior Scientist
Role Summary

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 AAV- mediated gene transfer approaches while developing treatments for rare genetically defined diseases that focus on the CNS, neuromuscular, and liver (systemic and metabolic) therapeutic areas. The Senior Scientist will be part of a larger interdisciplinary team to advance Pfizer's gene therapy programs with a focus on metabolic diseases. The role requires knowledge and experience performing in vivo experiments to evaluate AAV vectors and functionality. The Senior Scientist is expected to work independently on developing projects using AAV-directed gene therapy targeting metabolic disorders. The Senior Scientist is expected to develop and perform cell based and biochemical assays to characterize rAAV vectors in addition to developing in vivo disease models. The role requires having a deep knowledge of human disease, nucleic acid (oligo, DNA, siRNA/miRNA library) design/evaluation, and AAV biology. The candidate is expected to work independently contributing to development of new projects while progressing existing programs.

Role Responsibilities

• Design and screen nucleic acid sequences targeting both DNA and RNA; oligo vectorization, siRNA/miRNA library design, screening and vectorization

• Independently develop in vivo models and cell based assays to evaluate AAV gene therapy vectors targeting metabolic disorders
• Design, perform and investigate in vivo studies in rodents and non-human primates.
• Develop and perform routine cell based and biochemical assays supporting various programs
• Partner with colleagues working on Gene Therapy projects within the rare disease and other organizations


• Ph.D. in the life sciences with 2+ years of relevant experience in the field of metabolic disorders, nucleic acid library design/screening and AAV vectors.
• Prior experience with AAV vectors and direct hands on experience with 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:
  • Last Date to Apply: July 26, 2021
  • Eligible for Relocation Package
  • Eligible for Employee Referral Bonus

Sunshine Act

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EEO & Employment Eligibility

Pfizer is committed to equal opportunity in the terms and conditions of employment for all employees and job applicants without regard to race, color, religion, sex, sexual orientation, age, gender identity or gender expression, national origin, disability or veteran status. Pfizer also complies with all applicable national, state and local laws governing nondiscrimination in employment as well as work authorization and employment eligibility verification requirements of the Immigration and Nationality Act and IRCA. Pfizer is an E-Verify employer.

Research and Development


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