111 Gene Therapy jobs in the United States

Our client is a pioneering biotechnology firm at the cutting edge of genetic medicine, committed to developing novel gene therapies for severe diseases. We are seeking an exceptional Lead Gene Therapy Scientist to spearhead our research and development efforts. This role is pivotal in designing and executing experimental strategies for the development of advanced gene therapy vectors and therapeutic constructs. You will lead a team of talented scientists, driving projects from concept through preclinical stages. This is a fully remote position, allowing for global collaboration and innovation.

Responsibilities:

• Lead the design, development, and optimization of gene therapy vectors (e.g., AAV, lentivirus) and therapeutic payloads.
• Develop and validate robust in vitro and in vivo preclinical models to assess the efficacy and safety of gene therapy candidates.
• Manage and mentor a team of research associates and scientists, fostering a collaborative and high-performance research environment.
• Plan and oversee experimental workflows, ensuring rigorous scientific methodology and data integrity.
• Analyze complex biological data, interpret results, and draw sound conclusions regarding therapeutic potential.
• Collaborate with cross-functional teams, including vector manufacturing, pharmacology, toxicology, and regulatory affairs, to advance gene therapy programs.
• Stay abreast of the latest advancements in gene therapy, molecular biology, and related fields through literature review and conference attendance.
• Contribute to the preparation of scientific publications, presentations, and intellectual property filings.
• Oversee budget and resource allocation for assigned research projects.
• Ensure adherence to all safety, ethical, and regulatory guidelines within the research setting.

Qualifications:

• Ph.D. in Molecular Biology, Virology, Genetics, Bioengineering, or a related discipline.
• Minimum of 7-10 years of relevant research experience, with a significant focus on gene therapy vector development and/or preclinical evaluation.
• Demonstrated leadership experience in managing scientific projects and teams.
• In-depth knowledge of gene delivery systems, molecular cloning techniques, and genetic engineering principles.
• Proven experience with various gene therapy vector platforms (e.g., AAV serotypes, lentiviral vectors).
• Strong understanding of disease biology and the application of gene therapy.
• Hands-on experience with cell culture, molecular biology assays (PCR, qPCR, Western blot, ELISA), and potentially animal studies.
• Excellent analytical, problem-solving, and critical thinking skills.
• Exceptional written and verbal communication and presentation skills.
• Proven ability to thrive in a remote, fast-paced, and highly collaborative research environment.

This is a unique opportunity to drive innovation in gene therapy and make a profound impact on patient lives.

Our client is at the forefront of biotechnology innovation and is seeking an exceptional Lead Gene Therapy Researcher to join their pioneering, fully remote team. This is a unique opportunity to shape the future of medicine from anywhere in the country, working on groundbreaking therapies that target rare genetic disorders. You will lead a virtual team of scientists, driving research initiatives from conceptualization through to pre-clinical development. The ideal candidate will possess an extensive background in molecular biology, virology, and gene editing technologies, coupled with a strong understanding of therapeutic vector design and delivery. Your responsibilities will encompass designing and overseeing complex research projects, interpreting intricate biological data, and contributing to the strategic direction of the company's gene therapy pipeline. You will be expected to foster a collaborative and innovative remote work environment, actively engaging with team members through virtual platforms. This role requires a visionary leader with a passion for scientific discovery, exceptional problem-solving capabilities, and the ability to translate complex scientific concepts into actionable research plans. Your contributions will be critical in advancing novel treatments that have the potential to transform patient outcomes.

Key Responsibilities:

• Lead and manage research projects focused on the development of novel gene therapies.
• Design and optimize gene delivery vectors, including viral and non-viral systems.
• Develop and validate preclinical models to assess the efficacy and safety of gene therapies.
• Analyze and interpret complex genetic and biological data using advanced bioinformatics tools.
• Stay current with the latest advancements in gene therapy, genomics, and related fields.
• Mentor and guide remote research staff, fostering a high-performing team culture.
• Prepare research reports, regulatory documents, and scientific publications.
• Collaborate effectively with internal teams and external partners in a virtual setting.
• Contribute to the intellectual property strategy and patent applications.

Qualifications:

• Ph.D. in Molecular Biology, Genetics, Virology, Bioengineering, or a related discipline.
• Minimum of 8 years of progressive research experience in gene therapy or a closely related field.
• Demonstrated leadership experience in managing research projects and teams.
• Extensive knowledge of gene editing technologies (e.g., CRISPR), vectorology, and therapeutic delivery strategies.
• Proven ability to design and execute complex experiments and interpret data.
• Strong publication record in high-impact journals.
• Excellent communication, collaboration, and presentation skills for remote team engagement.
• Experience with bioinformatics and computational biology tools.
• Familiarity with regulatory requirements for gene therapy development.
• Ability to work independently and drive projects forward in a remote environment.

**Job Title:** Gene Therapy - Summer 2026 Intern
**Location:** Waltham, MA
**About the Job**
Are you ready to shape the future of medicine? The race is on to speed up drug discovery and development to find answers for patients and their families. Your skills could be critical in helping our teams accelerate progress.
Our team is looking for a summer intern to help in the development of new method(s) using modern chromatography techniques or evaluation of a new technology platform for its future implementation in the QC lab.
This individual shall work with different gene/cell therapy modalities such as AAV and LNP and apply various separation science platforms (HPLC/UPLC - Reverse phase, SEC and IEX with detections like UV, FLD, CAD and QDa; CE; cIEF; GC) or help the group deploy a novel/new platform in the lab.
We are an innovative global healthcare company with one purpose: to chase the miracles of science to improve people's lives. We're also a company where you can flourish and grow your career, with countless opportunities to explore, make connections with people, and stretch the limits of what you thought was possible. Ready to get started?
**Main Responsibilities:**
+ Support HPLC analytical method development for gene therapy modalities (AAV or LNP-mRNA).
+ Support new analytical technology assessment for gene therapy modalities (AAV or LNP-mRNA).
+ Support HPLC operation and maintenance.
+ Support compiling of historical data of Sanofi GMU AD/QC and trending analysis.
**About You**
**Basic Qualifications:**
+ Currently enrolled and pursuing bachelor's or master's degree in chemistry, biology, biochemistry, or related field at an accredited college or university with the expectation that you will complete your current degree by the Spring of 2027.
+ Must be enrolled in school the semester following your internship/co-op with Sanofi.
+ Must be able to relocate to the office location and work 40hrs/week, Monday-Friday, for the full duration of the co-op/internship.
+ **Must be permanently authorized to work in the U.S. and not require sponsorship of an employment visa (e.g., H-1B or green card) at the time of application or in the future. Students currently on CPT, OPT, or STEM OPT usually require future sponsorship for long term employment and do not meet the requirements for this program unless eligible for an alternative long-term status that does not require company sponsorship.**
**Preferred Qualifications:**
+ Good knowledge and skills in a biologic research lab setting and environment.
+ Enthusiasm for scientifically demanding challenges.
+ Interest in research and development in pharma and biotech industry.
+ Good written and spoken communication skills.
**Why Choose Us?**
+ Bring the miracles of science to life alongside a supportive, future-focused team.
+ Discover endless opportunities to grow your talent and drive your career, whether it's through a promotion or lateral move, at home or internationally.
+ Enjoy a thoughtful, well-crafted rewards package that recognizes your contribution and amplifies your impact.
+ Exposure to cutting-edge technologies and research methodologies.
+ Networking opportunities within Sanofi and the broader biotech community.
Sanofi Inc. and its U.S. affiliates are Equal Opportunity and Affirmative Action employers committed to a culturally diverse workforce. All qualified applicants will receive consideration for employment without regard to race; color; creed; religion; national origin; age; ancestry; nationality; marital, domestic partnership or civil union status; sex, gender, gender identity or expression; affectional or sexual orientation; disability; veteran or military status or liability for military status; domestic violence victim status; atypical cellular or blood trait; genetic information (including the refusal to submit to genetic testing) or any other characteristic protected by law.
#GD-SA
#LI-SA
#LI-Onsite
#vhd
**Pursue** **_progress_** **, discover** **_extraordinary_**
Better is out there. Better medications, better outcomes, better science. But progress doesn't happen without people - people from different backgrounds, in different locations, doing different roles, all united by one thing: a desire to make miracles happen. So, let's be those people.
At Sanofi, we provide equal opportunities to all regardless of race, colour, ancestry, religion, sex, national origin, sexual orientation, age, citizenship, marital status, ability or gender identity.
Watch our ALL IN video ( and check out our Diversity Equity and Inclusion actions at sanofi.com ( !
_US and Puerto Rico Residents Only_
Sanofi Inc. and its U.S. affiliates are Equal Opportunity and Affirmative Action employers committed to a culturally inclusive and diverse workforce. All qualified applicants will receive consideration for employment without regard to race; color; creed; religion; national origin; age; ancestry; nationality; natural or protective hairstyles; marital, domestic partnership or civil union status; sex, gender, gender identity or expression; affectional or sexual orientation; disability; veteran or military status or liability for military status; domestic violence victim status; atypical cellular or blood trait; genetic information (including the refusal to submit to genetic testing) or any other characteristic protected by law.
Global Terms & Conditions and Data Privacy Statement ( is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe. Discover more about us visiting or via our movie We are Sanofi ( an organization, we change the practice of medicine; reinvent the way we work; and enable people to be their best versions in career and life. We are constantly moving and growing, making sure our people grow with us. Our working environment helps us build a dynamic and inclusive workplace operating on trust and respect and allows employees to live the life they want to live.
All in for Diversity, Equity and Inclusion at Sanofi - YouTube (

Our client is a pioneering biotechnology firm committed to advancing cutting-edge therapies, and they are seeking an exceptional Lead Cell & Gene Therapy Scientist to join their fully remote research team. This role is instrumental in driving innovation and development within the rapidly evolving fields of cell and gene therapy. You will be responsible for leading research programs, developing novel therapeutic modalities, and contributing to the translation of promising discoveries into clinical applications. The ideal candidate will possess a Ph.D. in Molecular Biology, Immunology, Genetics, or a related field, with a minimum of 8 years of experience specifically focused on cell and gene therapy development. Proven expertise in areas such as viral vector design and production, CRISPR/Cas9 technology, CAR-T cell therapy development, or other advanced gene editing techniques is essential. You will oversee experimental design, guide scientific execution, and mentor a team of highly skilled researchers. Key responsibilities include troubleshooting complex scientific challenges, analyzing and interpreting experimental data, and ensuring rigorous scientific standards are maintained. Collaboration with internal teams (including manufacturing, regulatory affairs, and preclinical development) and external academic and industry partners will be crucial. A strong publication record, patent filings, and a deep understanding of the regulatory landscape for cell and gene therapies are highly valued. This is a 100% remote position, requiring exceptional communication, leadership, and self-management skills. If you are driven by the opportunity to shape the future of medicine and lead impactful research from a remote setting, we encourage you to apply.

Responsibilities:

• Lead and execute research projects in cell and gene therapy.
• Develop and optimize novel gene editing and cell engineering strategies.
• Oversee the design and execution of experimental studies.
• Analyze complex biological and genetic data.
• Mentor and guide junior scientists and research associates.
• Collaborate with cross-functional teams, including manufacturing and regulatory affairs.
• Troubleshoot research challenges and develop innovative solutions.
• Contribute to the development of intellectual property (patents).
• Stay current with advancements in cell and gene therapy technologies.
• Present research findings internally and externally.

Qualifications:

• Ph.D. in Molecular Biology, Immunology, Genetics, or a related field.
• 8+ years of experience in cell and gene therapy research and development.
• Specific expertise in areas such as gene editing (CRISPR), viral vector technology, or cellular immunotherapy (e.g., CAR-T).
• Strong understanding of molecular biology, genetics, and immunology.
• Proven ability to design, execute, and interpret complex experiments.
• Experience leading research teams and projects.
• Excellent written and verbal communication skills.
• Strong problem-solving and analytical abilities.
• Experience working effectively in a remote, collaborative environment.
• Publication record in high-impact journals is a plus.

Our client, a cutting-edge pharmaceutical company dedicated to developing life-changing gene therapies, is seeking a highly accomplished Principal Bioinformatics Scientist to join our innovative, fully remote research team. This critical role will lead the development and application of advanced computational and statistical methods to analyze complex biological data, driving the discovery and development of novel gene-based medicines. You will play a key part in interpreting genomic, transcriptomic, and epigenomic data from preclinical models and clinical studies, contributing to strategic decision-making and therapeutic advancement. As a remote-first employee, you will collaborate seamlessly with a global team of scientists, engineers, and clinicians, leveraging state-of-the-art tools and platforms.

Responsibilities:

• Lead the design and implementation of bioinformatics pipelines for the analysis of high-throughput sequencing data (e.g., WGS, WES, RNA-Seq, ChIP-Seq) relevant to gene therapy research.
• Develop and apply advanced statistical and machine learning models to identify potential drug targets, biomarkers, and mechanisms of action.
• Collaborate closely with molecular biologists, geneticists, and clinical scientists to define research questions and translate biological hypotheses into computational analyses.
• Interpret complex datasets, generate actionable insights, and effectively communicate findings to both technical and non-technical audiences through presentations and written reports.
• Stay at the forefront of bioinformatics and computational biology, evaluating and adopting new technologies and methodologies.
• Contribute to the development of our internal data infrastructure and knowledge management systems.
• Mentor junior bioinformatics staff and foster a collaborative, knowledge-sharing environment.
• Ensure reproducibility and rigor in all computational analyses.
• Engage with external collaborators, academic institutions, and key opinion leaders in the field.
• Contribute to regulatory submissions by providing robust bioinformatic analyses and documentation.

This fully remote position offers an exceptional opportunity to work on pioneering gene therapy programs targeting rare genetic disorders and other unmet medical needs. You will have access to significant computational resources and a supportive scientific environment. The ideal candidate possesses a Ph.D. in Bioinformatics, Computational Biology, Statistics, or a related quantitative field, with a minimum of 7-10 years of progressive experience in bioinformatics, preferably within the pharmaceutical or biotechnology industry. Demonstrated expertise in analyzing large-scale genomic and transcriptomic data, proficiency in programming languages (e.g., Python, R), and experience with cloud computing platforms (e.g., AWS, GCP) are essential. A strong understanding of gene therapy principles and disease mechanisms is highly valued. We are looking for a strategic thinker with excellent problem-solving skills and the ability to lead complex analytical projects from conception to completion. Your work will directly contribute to the development of transformative therapies for patients worldwide.

Our client, a leading biopharmaceutical research organization, is seeking an exceptional Principal Research Scientist to spearhead groundbreaking work in Gene Therapy at their state-of-the-art facility in **Baltimore, Maryland, US**. This senior position offers the opportunity to lead innovative research projects, mentor a team of scientists, and significantly contribute to the development of novel therapeutic solutions for unmet medical needs. The successful candidate will drive the scientific direction, experimental design, and execution of complex research programs focused on gene editing, vector development, and preclinical studies. Responsibilities include:

• Leading the design, execution, and interpretation of experiments in gene therapy research, utilizing cutting-edge molecular biology and cell biology techniques.
• Developing and optimizing novel gene delivery systems (e.g., viral vectors, non-viral methods) for therapeutic applications.
• Conducting in vivo and in vitro studies to evaluate the efficacy and safety of gene therapy candidates.
• Collaborating with cross-functional teams, including discovery, translational science, and CMC, to advance pipeline programs.
• Managing project timelines, resources, and budgets effectively to achieve research milestones.
• Mentoring and guiding junior scientists, fostering a collaborative and scientifically rigorous research environment.
• Analyzing complex datasets, drawing insightful conclusions, and presenting findings at internal meetings and external scientific conferences.
• Staying current with the latest advancements in gene therapy, genetic engineering, and related fields through literature review and scientific engagement.
• Contributing to patent applications and scientific publications.

The ideal candidate will possess a Ph.D. in Molecular Biology, Genetics, Biochemistry, or a related life science discipline, with a minimum of 8-10 years of post-doctoral and industry experience focused on gene therapy. A strong publication record demonstrating expertise in gene editing technologies, AAV or lentiviral vectorology, and preclinical models is essential. Demonstrated success in leading research projects and managing scientific teams is required. Excellent communication, presentation, and interpersonal skills are necessary to effectively collaborate with colleagues and present complex scientific information. This role is based in our **Baltimore, Maryland, US** research campus and requires full-time, on-site presence.

Our client, a cutting-edge biotechnology firm at the forefront of innovative therapeutic development, is seeking a highly accomplished Principal Scientist to lead critical research initiatives in Gene Therapy. This hybrid role is based in our state-of-the-art research facility in **Salt Lake City, Utah, US**, offering a dynamic work environment with opportunities for both collaborative in-lab work and focused independent research. You will be responsible for driving research strategies, designing and executing complex experiments, and contributing to the discovery and development of novel gene-based therapies. This position requires a deep understanding of molecular biology, genetics, and advanced therapeutic modalities.

Key responsibilities include designing and overseeing preclinical research programs focused on gene therapy delivery systems, vector engineering, and therapeutic efficacy. You will lead a team of research associates and scientists, providing mentorship, technical guidance, and fostering a collaborative research environment. The Principal Scientist will be responsible for data analysis, interpretation, and the preparation of high-quality scientific reports, publications, and presentations for internal stakeholders and external scientific forums. You will manage project timelines, budgets, and resources effectively. Collaborating with cross-functional teams, including process development, pharmacology, and regulatory affairs, to advance programs through the development pipeline is essential. Identifying and evaluating new technologies and scientific opportunities in gene therapy will be a key aspect of the role.

The ideal candidate will possess a Ph.D. in Molecular Biology, Genetics, Biochemistry, or a related field, with a strong publication record. A minimum of 8-10 years of progressive post-doctoral research experience in gene therapy or a closely related area, with demonstrated experience in leading research projects and teams, is required. Expertise in adeno-associated virus (AAV) vector design and production, gene editing technologies (e.g., CRISPR), and relevant biological assays is mandatory. Strong experience in preclinical study design and execution, including in vivo models, is essential. Excellent analytical, critical thinking, and problem-solving skills are required. Outstanding written and verbal communication skills, with the ability to present complex scientific information clearly and effectively, are critical. Experience with bioinformatics and computational biology tools is a plus. This is an unparalleled opportunity for a visionary scientist to make a significant impact in the field of gene therapy.

Our client, a groundbreaking leader in scientific research and development, is actively seeking a highly accomplished Principal Scientist to spearhead their gene therapy research initiatives. This is a fully remote position, offering a unique opportunity to contribute to pioneering work from the comfort of your home office, contributing to advancements based out of Virginia Beach, Virginia, US . You will lead innovative projects focused on developing novel gene-editing technologies and therapeutic approaches for rare genetic disorders.

Key Responsibilities:

• Lead the design and execution of cutting-edge research projects in gene therapy and gene editing.
• Develop and optimize novel viral and non-viral delivery vectors for therapeutic gene transfer.
• Investigate the efficacy and safety of gene therapy candidates in relevant preclinical models.
• Mentor and guide a team of scientists and research associates, fostering a collaborative and innovative research environment.
• Analyze and interpret complex experimental data, and translate findings into actionable research strategies.
• Stay abreast of the latest advancements in gene therapy, molecular biology, genetics, and related fields.
• Author high-impact scientific publications and present research findings at international conferences.
• Collaborate closely with cross-functional teams, including bioinformatics, pharmacology, and regulatory affairs.
• Contribute to the strategic direction of the gene therapy pipeline and research programs.
• Manage external collaborations with academic institutions and research partners.
• Ensure compliance with all laboratory safety standards and ethical research practices.
• Lead the development of new research methodologies and technologies.
• Manage research budgets and resource allocation for assigned projects.

The ideal candidate will possess a Ph.D. in Molecular Biology, Genetics, Biochemistry, or a related field, with a distinguished record of scientific achievement and publications in peer-reviewed journals. A minimum of 8 years of relevant post-doctoral and/or industry experience in gene therapy, gene editing (e.g., CRISPR), or nucleic acid therapeutics is essential. Extensive hands-on experience with vector design and production (AAV, lentivirus), cell-based assays, and preclinical model development is required. Proven leadership experience, with the ability to mentor and manage a team of scientists, is crucial. Exceptional analytical, problem-solving, and critical thinking skills are necessary. Outstanding written and verbal communication skills are vital for presenting complex scientific information and collaborating effectively. Experience with rare disease indications is a strong plus. Join our client and make a significant impact on the future of genetic medicine.