Senior Scientist, In Vivo Gene Editing & Hematology

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

Benefits offered by this job

Annual bonus

Equity awards

Generous paid time off

Job summary

Vertex Pharmaceuticals LLC seeks an innovative physician-scientist to lead drug discovery and translational research within the Hemoglobinopathies Research Group. The role focuses on advancing in vivo gene editing programs from preclinical proof-of-concept through IND-enabling studies and into Phase 1/2 trials.

The successful candidate will bridge clinical expertise with translational science, shape disease strategy, direct preclinical and translational research, and contribute across a broader

Qualifications

• MD and/or PhD with 11+ years in medicine or biotech translational R&D.
• Strong expertise in drug development and disease biology.
• Experience in cell and gene therapy, genome editing, or in vivo pharmacology.
• Track record in hemoglobinopathies and translational therapeutics.

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

Scientific leadership

Translational research

Drug development

Gene therapy

Hematology expertise

Cross-functional leadership

Education

MD or PhD in a relevant discipline

Job description

Vertex Pharmaceuticals LLC seeks an innovative physician-scientist to lead drug discovery and translational research within the Hemoglobinopathies Research Group. The role focuses on advancing in vivo gene editing programs from preclinical proof-of-concept through IND-enabling studies and into Phase 1/2 trials.

The successful candidate will bridge clinical expertise with translational science, shape disease strategy, direct preclinical and translational research, and contribute across a broader