Research Scientist I -The Center for Therapeutic Genetics (CTG)

OVERVIEW

The mission of the Broad Institute of MIT and Harvard is to propel the understanding and treatment of disease. We apply our deep knowledge of human genetics to empower a revolution in biomedicine and accelerate the pace at which the world conquers disease. Through our partnerships with MIT, Harvard, and Harvard-affiliated hospitals, we’ve become a global hub of cutting-edge biomedical science.

The Center for Therapeutic Genetics (CTG) is advancing a platform-based approach to precision genetic medicine, translating CRISPR-based nucleases, base editors, prime editors, and other genome-modifying technologies into IND-enabling and clinical-stage programs for rare disease. In this position, you will support the Nonclinical Team Lead in the strategic design, execution, and regulatory advancement of CTG's nonclinical development pipeline, contributing hands-on scientific expertise in genome editing technology development and specificity profiling across CTG therapeutic programs, including Alternating Hemiplegia of Childhood (AHC), Dravet syndrome, and future platform-enabled indications.

This role sits within CTG's nonclinical research team and works closely with the Nonclinical Team Lead, disease biologists, in vivo specialists, and external partners (e.g., The Jackson Laboratory, Boston Children's Hospital) to generate the editing efficacy, specificity, and technology-selection data that underpin platform-based IND strategy.

KEY RESPONSIBILITIES

- Design, execute, and analyze experiments evaluating precision genome editing technologies (e.g base editing and prime editing) for use across CTG therapeutic programs.
- Lead off-target and specificity profiling workstreams using genome-wide methods (CIRCLE-seq, ONE-seq, CHANGE-seq BE, and newly reported methods) to characterize candidates and inform go/no-go decisions.
- Design and execute high-throughput screens to identify, optimize, and prioritize editing strategies and guide/pegRNA designs for programs in neurologic and metabolic disease.
- Develop and analyze high-throughput-sequencing-based assays, including amplicon sequencing and library preparation, and interpret editing outcome and specificity data to support nonclinical data packages.
- Contribute scientific input to preclinical program strategy, including target and modality selection, editor design, and study design, in coordination with the Nonclinical Team Lead and disease biology leads.
- Evaluate emerging editing platforms and technologies, including those from external partners and collaborators, and provide technical assessments to inform partnership decisions.
- Support the generation of data supporting a platform-based IND paradigm, enabling reuse of specificity and safety data across multiple indications.
- Prepare clear, well-organized data summaries, figures, and written reports for internal review, sponsored research external reports, regulatory submissions, and FDA engagements.
- Collaborate with internal and external research partners, including other academic investigators and specialized service providers, to execute editing technology development and profiling studies.
- Maintain rigorous documentation and quality standards for experimental data, ensuring reproducibility and compliance with applicable internal and regulatory requirements.
- Stay current with the genome editing and gene therapy literature and proactively bring forward new technologies, methods, and competitive intelligence relevant to CTG programs.
- Mentor junior scientists, research associates, and trainees on genome editing and specificity profiling techniques as the team grows.

QUALIFICATIONS

- PhD in molecular biology, genetics, genome engineering, biomedical engineering, or a related field (0+ years of experience); or a Master’s degree in a related field with 5+ years of relevant industry/research experience; or a Bachelor’s degree in a related field with 7+ years of progressive, relevant industry experience.
- Proven track record of deep expertise in base editing and/or prime editing.
- Experience with unbiased genome-wide specificity profiling methods such as GUIDE-seq, Digenome-seq, CIRCLE-seq, ONE-seq, CHANGE-seq (or CHANGE-seq BE), PEG-seq, or related methods strongly preferred.
- Experience designing and executing high-throughput lentiviral screens strongly preferred.
- Proficiency in HTS library preparation and amplicon sequencing, with the ability to analyze and interpret sequencing data (computational collaboration or independent analysis).
- Familiarity with preclinical/IND-enabling study design and the translational path for gene editing therapeutics is a plus.
- Interest or prior experience in neurologic and metabolic disease biology is a plus.
- Strong record of rigorous, reproducible experimental work; publications and/or conference presentations preferred.
- Excellent written and verbal communication skills, including the ability to translate complex genome editing science for cross-functional and non-specialist audiences.
- Able to work collaboratively within a large, cross-functional, deadline-driven research group while also executing independently.
- Comfortable operating in a dynamic environment with multiple overlapping and shifting priorities.

The expected base pay range for this position as listed above is based on a 40 hour per week schedule. Broad provides pay ranges representing its reasonable and good faith estimate of what the organization reasonably expects to pay for a position at the time of posting. Actual compensation will vary based on factors including but not limited to, relevant skills, experience, education, qualifications, and other factors permissible by law. At Broad, your base pay is just one part of a comprehensive total rewards package. From day one, this role offers a competitive benefits package (https://www.broadinstitute.org/benefits/benefits-working-broad) including medical, dental, vision, life, and disability insurance; a 401(k) retirement plan; flexible spending and health savings accounts; at least 13 paid holidays; winter closure; paid time off; parental and family care leave; and an employee assistance program, among other Broad benefits.
The Broad Institute is an equal opportunity employer. All qualified applicants will receive consideration for employment without regard to race, national origin, religion, age, color, sex, disability, protected veteran status, or any other characteristic protected by local, state, or federal laws, rules, or regulations.
Should you need a reasonable accommodation to complete the application or interview process, please contact [email protected] for assistance.