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Scientist Gene Therapy Jobs (NOW HIRING)

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Scientist Gene Therapy information

What does a scientist in gene therapy do?

A Scientist in Gene Therapy researches and develops methods to treat genetic disorders by altering genes within a patient’s cells. Their work involves designing and conducting experiments, analyzing genetic data, and developing new gene-editing techniques such as CRISPR. They often collaborate with other scientists, clinicians, and regulatory agencies to advance therapies from the lab to clinical trials. These scientists play a critical role in discovering treatments for diseases like cystic fibrosis, sickle cell anemia, and certain cancers.

What are some common challenges faced by a scientist in gene therapy when translating research from the lab to clinical applications?

One of the primary challenges Gene Therapy Scientists encounter is ensuring that promising laboratory results translate effectively and safely into clinical settings. This involves navigating complex regulatory requirements, scaling up vector production while maintaining quality, and addressing potential immune responses in human subjects. Collaboration with clinicians, regulatory experts, and manufacturing teams is essential to troubleshoot issues and optimize protocols. Staying current with evolving technologies and guidelines also plays a key role in successfully advancing therapies toward patient use.

What are the key skills and qualifications needed to thrive as a scientist in gene therapy, and why are they important?

To thrive as a Scientist in Gene Therapy, you need expertise in molecular biology, cell culture, and genetic engineering, typically backed by an advanced degree (PhD or MS) in life sciences. Proficiency with CRISPR/Cas9, viral vector technologies, PCR, and bioinformatics software is often required. Strong analytical thinking, problem-solving skills, and the ability to communicate complex data effectively help you excel in this field. These skills and qualities are crucial for developing safe and effective gene therapies, meeting regulatory standards, and advancing scientific innovation.

What is the difference between Scientist Gene Therapy vs Research Scientist?

AspectScientist Gene TherapyResearch Scientist
Required CredentialsMaster's or PhD in Molecular Biology, Genetics, or related fieldBachelor's or higher in a scientific discipline
Work EnvironmentLaboratories focused on gene therapy development, clinical research settingsVarious research settings including academia, industry, labs
Employer & Industry UsageBiotech, pharmaceutical companies, clinical research organizationsUniversities, research institutes, industry

Scientist Gene Therapy and Research Scientist roles often share similar educational backgrounds and work environments. However, Scientist Gene Therapy specializes in developing and testing gene therapy treatments, whereas Research Scientists may work across diverse scientific fields. Both roles are vital in advancing medical research, but the focus and specific expertise differ.

What cities are hiring for Scientist Gene Therapy jobs?

Cities with the most Scientist Gene Therapy job openings:

What states have the most Scientist Gene Therapy jobs?

States with the most job openings for Scientist Gene Therapy jobs include:

What are popular job titles related to Scientist Gene Therapy jobs?

For Scientist Gene Therapy jobs, the most frequently searched job titles are:

Infographic showing various Scientist Gene Therapy job openings in the United States as of September 2026, with employment types broken down into 100% Full Time. Highlights an 100% In-person job distribution.

Postdoctoral Scientist - Gene Therapy Biodistribution & Molecular Biology (CNS)

Boston, MA • On-site

Eli Lilly and Company
Pharmaceutical Product Wholesalers • 10K+ employees

Full-time

Medical, Dental, Vision, Life, Retirement, PTO

Re-posted 12 days ago


Key responsibilities

  • Design, develop, optimize, and execute biodistribution assays to assess gene therapy vector distribution and transgene expression in CNS and peripheral tissues.

  • Independently design and troubleshoot CNS gene therapy biodistribution studies, ensuring data quality and experimental rigor.

  • Interface with external CROs to oversee in vivo biodistribution studies and ensure scientific standards are met.


Eli Lilly and Company rating

8.9

Company rating: 8.9 out of 10

Based on 64 frontline employees who took The Breakroom Quiz

9th of 86 rated pharmaceutical


Job description

At Lilly, the work is demanding because patients are waiting. We unite caring with discovery to help make life better for people around the world, knowing that every decision, every detail, and every day matters. Headquartered in Indianapolis, Indiana, our over 50,000 employees around the globe take on complex challenges to discover and deliver life-changing medicines, strengthen how health is understood and managed, and support the communities we serve. This is hard, urgent, selfless work-but it's work worth doing. If you're driven by purpose and ready to bring your best to work that truly matters for patients, we invite you to join us.


Position Overview:

Eli Lilly and Company is seeking a highly motivated Postdoctoral Scientist to join the Drug Delivery and Connected Systems (DDCS) organization, with a primary focus on CNS gene therapy biodistribution and molecular bioanalysis. This role will support the optimization and translational advancement of gene therapy platforms, both viral and non-viral, for neurological and neurodegenerative diseases.

The successful candidate will bring deep expertise in biodistribution assay development, molecular biology, and quantitative bioanalysis, and will work at the interface of in vivo CNS pharmacology, vector biology, and translational science. This position offers hands-on involvement in preclinical CNS gene therapy programs, collaboration with internal cross-functional teams, and scientific oversight of external CRO-led studies.

This postdoctoral role offers a unique opportunity to contribute directly to cutting-edge CNS gene therapy programs within a leading pharmaceutical research organization. The position provides high-impact scientific exposure, cross-functional collaboration, and skill development at the intersection of gene therapy biology, biodistribution science, and translational CNS drug delivery. There will be opportunity to publish in peer reviewed journals.

Key Responsibilities:

Biodistribution & Molecular Bioanalysis

  • Lead the design, development, optimization, and execution of biodistribution assays to assess AAVs, LNPs, siRNA distribution, transgene expression, and persistence across CNS and peripheral tissues.

  • In depth knowledge of vector biology, and AAV serotype-specific biodistribution knowledge (eg: AAV9, AAVrh10, PHP.eB) is a must- CNS tropism and off-target profiles are serotype-dependent and fundamental to interpreting results.

  • Apply a broad toolkit of molecular and bioanalytical techniques, including:PCR-based approaches (PCR, qPCR, ddPCR, stem-loop PCR, vg/dg dose quantification) for vector genome and transcript quantification; ELISA and immunoassays for protein expression and biomarker analysis; LC-MS-based quantitative bioanalysis where applicable; Immunohistochemistry (IHC) and tissue-based spatial analyses to enable regional CNS characterization; Next-generation sequencing (NGS) methods to support vector tracking, expression profiling, or integration-related analyses

  • Ensure assays are fit-for-purpose, sensitive, reproducible, and aligned with translational gene therapy objectives.

  • Off-target analysis and DRG toxicology assay development.

Experimental Design, Troubleshooting, and Scientific Rigor

  • Independently design and execute CNS gene therapy-focused biodistribution studies, applying sound experimental design and Design of Experiments (DoE/DOR) principles.

  • Diagnose and troubleshoot complex technical challenges related to vector detection, tissue processing, assay performance, or data variability.

  • Maintain meticulous experimental documentation, ensuring clear traceability, data integrity, and compliance with DDCS best practices.

CNS Gene Therapy & Translational Insight

  • Apply strong foundational knowledge of CNS biology, molecular biology, and biochemistry to interpret biodistribution and expression data in a therapeutic context.

  • Integrate biodistribution results with an understanding of pharmacokinetics and pharmacodynamics (PK/PD) to inform dose selection, delivery route optimization, and therapeutic index assessment for viral and non-viral gene therapies.

  • Contribute to translational strategy discussions by linking molecular readouts to functional, anatomical, and disease-relevant endpoints.

In Vivo Study Oversight & External Collaboration

  • Independently interface with external CROs to design, execute, and oversee in vivo gene therapy biodistribution studies, including work in higher vertebrate CNS models.

  • Provide scientific oversight for tissue collection, sample processing, and downstream molecular analyses conducted internally or externally.

  • Ensure CRO outputs meet DDCS scientific standards, timelines, and data-quality expectations.

Data Analysis, Visualization, and Team Communication

  • Apply best practices for managing and analyzing large, multi-dimensional biodistribution datasets generated from CNS gene therapy studies.

  • Perform statistical analysis and generate clear, insightful data visualizations to support project teams and decision-making forums.

  • Communicate findings effectively through presentations, written summaries, and collaborative scientific discussions.

  • Actively contribute as a proactive, team-oriented scientist within a highly collaborative DDCS research environment.

Basic Requirements:

  • PhD in Molecular Biology, Biochemistry, Pharmacology, Neuroscience, Bioengineering, or a closely related life sciences discipline.

  • Demonstrated in-depth experience in AAV biodistribution analysis and/or molecular bioanalytical assay development.

  • Strong hands-on expertise in PCR/qPCR/ddPCR, ELISA, IHC, NGS, and/or related molecular biology techniques.

  • Solid understanding of CNS gene therapy delivery, molecular mechanisms of transgene expression, and/or tissue-specific biology.

  • Proven ability to independently design experiments, troubleshoot technically challenging assays, and maintain rigorous scientific documentation.

  • Highly motivated, proactive, and able to thrive in a collaborative, team-based research environment.

  • Qualified applicants must be authorized to work in the United States on a full-time basis. Lilly will not provide support for or sponsor work authorization or visas for this role, including but not limited to F-1 CPT, F-1 OPT, F-1 STEM OPT, J-1, H-1B, TN, O-1, E-3, H-1B1, or L-1.


Additional Preferences:

  • Knowledge of pharmacokinetics and pharmacodynamics as applied to gene therapy biodistribution and expression.

  • Spatial transcriptomics / RNAscope as a preferred qualification for CNS transgene expression mapping.

  • Direct experience working with CNS tissues and CNS-relevant animal models for gene therapy research.

  • Experience integrating biodistribution data with imaging, functional, or efficacy readouts.

  • Experience collaborating with CROs and supporting in vivo gene therapy studies.

  • Familiarity with FDA (2020) long-term follow-up guidance and EMA biodistribution reflection papers as preferred qualifications. Knowledge of current FDA guidance on intrathecal/systemic AAV safety signals.

Other Information:

  • This is a fixed-term role for one-year, extendable up to four years, with eligibility to apply for full-time positions upon completion.

  • This position is based out of the Boston, MA Lilly location, and requires daily, on-site presence and lab work.

About Eli Lilly's Postdoctoral Scientist Program

When it comes to research and development, our goal is to discover and deliver innovative medicines that make life better for people around the world. It's challenging, expensive and often filled with failure. But even when we fail, we advance medical science and understanding by learning more about diseases, biology and chemistry - ultimately bringing new solutions one step closer to reality.

Over the course of our history, we have shed light on some of the toughest health care problems known to man - diabetes, heart disease, infectious diseases, neuroscience disorders, cancer and more. We could not pursue this without our research and development team.

Postdoctoral scientists help us continue this pursuit. During your experience you will get:

  • Top industry research experience

  • Mentoring by some of Lilly's top scientists

  • Laboratory and classroom training and education to further your development

  • Collaboration and networking across dozens of postdoctoral scientists and other researchers

Lilly is dedicated to helping individuals with disabilities to actively engage in the workforce, ensuring equal opportunities when vying for positions. If you require accommodation to submit a resume for a position at Lilly, please complete the accommodation request form (https://careers.lilly.com/us/en/workplace-accommodation) for further assistance. Please note this is for individuals to request an accommodation as part of the application process and any other correspondence will not receive a response.


Lilly is proud to be an EEO Employer and does not discriminate on the basis of age, race, color, religion, gender identity, sex, gender expression, sexual orientation, genetic information, ancestry, national origin, protected veteran status, disability, or any other legally protected status.


Our employee resource groups (ERGs) offer strong support networks for their members and are open to all employees. Our current groups include: Africa, Middle East, Central Asia (AMECA), Black Employees at Lilly (BE@Lilly), Chinese Culture Network (CCN), EnAble, Evolve, Lilly Indian Network (LIN), Organization of Latinx at Lilly (OLA), Pride (LGBTQ+ Allies), Veterans Leadership Network (VLN) and Women's Initiative for Leading at Lilly (WILL).


Actual compensation will depend on a candidate's education, experience, skills, and geographic location. The anticipated wage for this position is

$58,000 - $123,200

Full-time equivalent employees also will be eligible for a company bonus (depending, in part, on company and individual performance). In addition, Lilly offers a comprehensive benefit program to eligible employees, including eligibility to participate in a company-sponsored 401(k); pension; vacation benefits; eligibility for medical, dental, vision and prescription drug benefits; flexible benefits (e.g., healthcare and/or dependent day care flexible spending accounts); life insurance and death benefits; certain time off and leave of absence benefits; and well-being benefits (e.g., employee assistance program, fitness benefits, and employee clubs and activities).Lilly reserves the right to amend, modify, or terminate its compensation and benefit programs in its sole discretion and Lilly's compensation practices and guidelines will apply regarding the details of any promotion or transfer of Lilly employees.

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About Eli Lilly

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Eli Lilly, based in Indianapolis, IN, US, is one of the pioneers in the pharmaceutical industry with a rich history dating back to 1876. This global pharmaceutical company focuses on discovering, developing, manufacturing and selling pharmaceutical products in approximately 120 countries. The company's product categories include endocrinology, oncology, cardiovascular, neuroscience, and immunology. Having invested over $9 billion in research and development in the past decade, Eli Lilly is also committed to creating high-quality medicines that meet real needs. As a recipient of several awards and recognitions, Eli Lilly is known for its focus on life-saving research and drug development. Their mission is to make medicines that help people live longer, healthier, and more active lives.

Industry

Pharmaceutical product wholesalers

Company size

10,000+ Employees

Headquarters location

Indianapolis, IN, US

Year founded

1876