Lead, In Vivo Gene Editing Translational Scientist

Vertex Pharmaceuticals LLC; Boston, Northern (MA, KY); Hybrid; USD 197,000 - 296,000; Full time

Benefits offered by this job

Annual bonus

Equity awards

Comprehensive benefits

Job summary

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

The successful candidate will bridge clinical expertise with translational science, directing preclinical and translational research while shaping disease strategy and evidence-based

Qualifications

• MD and/or PhD in a relevant discipline with 11+ years in translational research or biotech/pharma R&D.
• Strong expertise in drug development and disease biology.
• Experience leading cross-functional teams and influencing program direction.

Responsibilities

• Lead scientific strategy for in vivo gene editing platforms and delivery approaches.
• Provide disease insights to support target selection and candidate optimization.
• Design and guide in vitro, ex vivo, and in vivo studies linked to clinically meaningful biology.
• Lead IND-enabling strategy with development, regulatory, and clinical teams.
• Manage cross-functional project teams, budgets, and decision points.
• Translate complex datasets into clear program recommendations.

Skills

Scientific leadership

Cross-functional collaboration

Drug development

Translational research

Education

MD/PhD

Job description

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

The successful candidate will bridge clinical expertise with translational science, directing preclinical and translational research while shaping disease strategy and evidence-based