Heme, Research Fellow

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

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

Pay Range

Pay Range: $197,400 - $296,200 Disclosure 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. 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

Designated 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 [e-mail].

Help Us Achieve Our Mission Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia, acute pain and acromegaly, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, congenital adrenal hyperplasia, ACTH-dependent Cushing's syndrome, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1.

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube, and X.

We believe our culture is one of Vertex’s greatest strengths and a key enabler of our ability to deliver transformative medicines for patients who need them. Rooted in innovation, purpose and collaboration, our culture empowers people to take smart risks, challenge conventional thinking and do their best work. The diversity of our people is central to that culture. By bringing together individuals with different backgrounds, experiences and perspectives, we foster an environment where new ideas thrive, better decisions are made and breakthrough innovation is possible. We are committed to creating a workplace where everyone feels valued, included and connected to our shared mission, because we know that our unique culture and the collective strength of our people are what set Vertex apart.