Translational Gene Editing Leader Hemoglobinopathies

Vertex Pharmaceuticals Inc (US); United States; On-site; USD 197,000 - 296,000; Full time

Benefits offered by this job

On-site work

Annual bonus

Job summary

Vertex Pharmaceuticals Inc (US) is seeking an innovative physician-scientist to lead drug discovery and translational research within the Hemoglobinopathies Research Group. The role drives in vivo gene editing programs from concept to IND-enabling studies and into early clinical trials, bridging clinical expertise with translational science across the Leiden portfolio and HSC biology initiatives.

The candidate will provide disease insight, guide cross-functional teams, and represent the

Qualifications

• MD and/or PhD in a relevant discipline
• Strong expertise in drug development and disease biology
• Demonstrated leadership and ability to influence project direction
• Experience in cell and gene therapy, genome editing, or in vivo pharmacology

Responsibilities

• Lead scientific strategy for in vivo gene editing platforms and delivery approaches
• Provide disease and mechanistic insight to support target selection and candidate optimization
• Design and guide in vitro, ex vivo, and in vivo studies tied to clinically meaningful biology
• Serve as physician-scientist leader for IND-enabling strategy with development, regulatory, and clinical teams
• Lead cross-functional project teams to set goals, timelines, budgets, and decision points
• Integrate scientific, translational, and clinical considerations to drive programs to development candidacy

Skills

MD/PhD in relevant discipline

Scientific leadership

Translational science

Genome editing

In vivo pharmacology

Education

MD and/or PhD in a relevant discipline

Job description

Vertex Pharmaceuticals Inc (US) is seeking an innovative physician-scientist to lead drug discovery and translational research within the Hemoglobinopathies Research Group. The role drives in vivo gene editing programs from concept to IND-enabling studies and into early clinical trials, bridging clinical expertise with translational science across the Leiden portfolio and HSC biology initiatives.

The candidate will provide disease insight, guide cross-functional teams, and represent the