1+ months

Principal Scientist, Translational Gene Therapy RNA therapeutics

Cambridge, MA 02139
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


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

**Pfizer requires all U.S. new hires to be fully vaccinated for COVID-19 prior to the first date of employment. As required by applicable law, Pfizer will consider requests for Reasonable Accommodations.**

**Sunshine Act**

Pfizer reports payments and other transfers of value to health care providers as required by federal and state transparency laws and implementing regulations. These laws and regulations require Pfizer to provide government agencies with information such as a health care provider's name, address and the type of payments or other value received, generally for public disclosure. Subject to further legal review and statutory or regulatory clarification, which Pfizer intends to pursue, reimbursement of recruiting expenses for licensed physicians may constitute a reportable transfer of value under the federal transparency law commonly known as the Sunshine Act. Therefore, if you are a licensed physician who incurs recruiting expenses as a result of interviewing with Pfizer that we pay or reimburse, your name, address and the amount of payments made currently will be reported to the government. If you have questions regarding this matter, please do not hesitate to contact your Talent Acquisition representative.

**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


Posted: 2021-08-02 Expires: 2021-11-10

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Principal Scientist, Translational Gene Therapy RNA therapeutics

Cambridge, MA 02139

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