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 cultureRequired 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 fieldsPreferred 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:$197,400 - $296,200Disclosure Statement:The range provided is based on what we believe is a reasonable estimate for the base salary pay range for this job at the time of posting. This role is eligible for an annual bonus and annual equity awards. Some roles may also be eligible for overtime pay, in accordance with federal and state requirements. Actual base salary pay will be based on a number of factors, including skills, competencies, experience, and other job-related factors permitted by law. At Vertex, our Total Rewards offerings also include inclusive market-leading benefits to meet our employees wherever they are in their career, financial, family and wellbeing journey while providing flexibility and resources to support their growth and aspirations. From medical, dental and vision benefits to generous paid time off (including a week-long company shutdown in the Summer and the Winter), educational assistance programs including student loan repayment, a generous commuting subsidy, matching charitable donations, 401(k) and so much more.Flex Designation:On-Site DesignatedFlex Eligibility Status:In this On-Site designated role, you will work five days per week on-site with ad hoc flexibility.
