Job Description

Position Summary

We are seeking an innovative physician-scientist to lead drug discovery and translational research within the Hemoglobinopathies Research Group. This role serves as the cross-functional Research Project Lead for in vivo gene editing therapies, with additional responsibilities across the Leiden Research portfolio and broader hematopoietic stem cell (HSC) biology initiatives.

The primary focus is to advance in vivo gene editing programs from preclinical proof-of-concept through IND-enabling studies and into Phase 1/2 trials. This leader will bridge clinical expertise with translational science to shape disease strategy, direct preclinical and translational research, and define the path to first-in-human studies.

The successful candidate will bring deep expertise in hemoglobinopathies, a strong translational mindset, and the ability to contribute across broader portfolio priorities where human disease biology, autoimmune relevance, and clinical development strategy intersect.

Key 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

Required 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

Preferred Experience

• Direct experience in hemoglobinopathies (e.g., sickle cell disease, thalassemia, erythropoiesis, globin regulation)
• Experience with IND-enabling programs and translation to first-in-human strategy
• Familiarity with delivery systems, editing efficiency, durability, tissue specificity, and off-target risk
• Knowledge of HSC biology, stem cell phenotyping, engraftment, and ex vivo manipulation
• Experience with conditioning strategies for cell/gene therapy or transplantation
• Familiarity with FDA/EMA regulatory pathways and GCP
• Ability to lead through expertise in a matrixed environment, communicate across disciplines, and mentor others

At Vertex, we embrace continuous learning and encourage employees to stay informed on advances in AI, technology, automation, and industry trends. We seek talent who bring an external perspective, demonstrate curiosity and sound judgment, and thoughtfully adopt new capabilities that improve decision-making, drive innovation, increase efficiency, and help deliver better outcomes for patients.

Pay Range:

Disclosure Statement:

Flex Designation:

Flex Eligibility Status:

In this On-Site designated role, you will work five days per week on-site with ad hoc flexibility.  

Note: The Flex status for this position is subject to Vertex’s Policy on Flex @ Vertex Program and may be changed at any time.

#LI-Onsite

Company Information

Vertex is a global biotechnology company that invests in scientific innovation.

Vertex is committed to equal employment opportunity and non-discrimination for all employees and qualified applicants without regard to a person's race, color, sex, gender identity or expression, age, religion, national origin, ancestry, ethnicity, disability, veteran status, genetic information, sexual orientation, marital status, or any characteristic protected under applicable law. Vertex is an E-Verify Employer in the United States. Vertex will make reasonable accommodations for qualified individuals with known disabilities, in accordance with applicable law.

Any applicant requiring an accommodation in connection with the hiring process and/or to perform the essential functions of the position for which the applicant has applied should make a request to the recruiter or hiring manager, or contact Talent Acquisition at [email protected]