Research Fellow: In Vivo Gene Editing & Translational Lead

Vertex Pharmaceuticals Incorporated; Boston (MA); On-site; USD 197,000 - 296,000; Full time

Job summary

Vertex Pharmaceuticals Incorporated seeks an innovative physician-scientist to lead drug discovery and translational research within the Hemoglobinopathies Research Group, focusing on in vivo gene editing programs from preclinical through IND-enabling studies and into Phase 1/2 trials.

The role bridges clinical expertise with translational science to shape disease strategy, direct preclinical and translational research, and define the path to first-in-human studies.

Qualifications

• MD and/or PhD in a relevant discipline with 11+ years in academic medicine, translational research, biotech, pharma R&D, or related settings
• Strong expertise in drug development, translational science, and disease biology
• Demonstrated scientific leadership, publication record, and ability to influence project direction
• Experience in cell and gene therapy, genome editing, or in vivo pharmacology
• Track record in hemoglobinopathies, translational therapeutics, gene therapy, gene editing, stem cell biology, or related fields

Responsibilities

• Lead scientific strategy for in vivo gene editing platforms, including editing technologies, delivery approaches, tissue targeting, and integrated pharmacology/toxicology
• Provide disease and mechanistic insight to support target selection, hypothesis testing, and candidate optimization
• Design and guide in vitro, ex vivo, and in vivo studies tied to clinically meaningful biology
• Serve as a physician-scientist leader for IND-enabling strategy in partnership with development, regulatory, and clinical teams
• Lead cross-functional project teams to set goals, priorities, timelines, budgets, and decision points
• Integrate scientific, translational, and clinical considerations to drive programs toward development candidate nomination
• Interpret complex datasets and convert findings into clear program recommendations
• Represent the organization externally through conferences, collaborations, publications, patents, and presentations
• Help build a strong, collaborative, high-performing research culture

Skills

Physician-scientist

Translational research

Drug development

Gene editing

Leadership

Education

MD or PhD

Tools

In vivo pharmacology

Genome editing

Job description

Vertex Pharmaceuticals Incorporated seeks an innovative physician-scientist to lead drug discovery and translational research within the Hemoglobinopathies Research Group, focusing on in vivo gene editing programs from preclinical through IND-enabling studies and into Phase 1/2 trials.

The role bridges clinical expertise with translational science to shape disease strategy, direct preclinical and translational research, and define the path to first-in-human studies.