Heme, Research Fellow

Vertex Inc.•Boston, MA

1d•$197,400 - $296,200•Onsite

About The Position

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.

Requirements

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

Nice To Haves

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

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