Scientist, Research Scientist, Biotech Research

Scientist Position

Seeking a Scientist to join a Research Company in Cambridge, MA. Role Overview

Our client is seeking an experienced, independent scientist to help drive the design, execution, and interpretation of studies supporting our engineered suppressor tRNA (sup-tRNA) pipeline in rare diseases. In this role, you will take ownership of research questions across multiple biological scales from molecular and biochemical characterization through in vivo efficacy and translational modeling. You will bring both the technical depth to execute complex studies and the judgment to design and troubleshoot experiments with minimal oversight, while working closely with a cross-functional team.

Key Responsibilities

• Independently designing, executing, and interpreting in vivo and in vitro studies evaluating AAV-delivered sup-tRNA gene therapy candidates, including dosing, tissue collection, and biodistribution and efficacy assessments.
• Leading the characterization of novel disease models through integrated molecular, biochemical, and functional analyses to inform go/no-go decisions.
• Applying quantitative molecular techniques (e.g., ddPCR, qPCR/RT-qPCR) to measure vector genomes, transgene and target protein expression, and biodistribution.
• Assessing protein-level readouts of therapeutic efficacy via JESS Western blot, ELISA, immunohistochemistry, MSD, or related methods.
• Contributing scientific strategy and study design input to IND-enabling packages, target product profiles, and regulatory or partnering documents.
• Presenting data and recommendations to the leadership team, translating complex datasets into clear conclusions.
• Collaborating cross-functionally with CMC, analytical development, and external CROs/CDMOs, and mentoring junior scientists as needed.
• Maintaining rigorous documentation and experimental records consistent with GLP-adjacent and IND-enabling study expectations.

Required Qualifications

Education:

A BS, MS or PhD in a biological or chemical discipline is required.

Experience:

Several years of relevant industry experience is necessary. Prior experience in the biotech or pharmaceutical industry, ideally within a gene therapy, rare disease, or drug discovery/development setting, is expected. A demonstrated track record of designing and independently executing experiments and studies is required.

Technical

Skills:

Hands-on expertise in molecular and protein-based assays (e.g., ddPCR/qPCR, Western blot, ELISA, IHC) is needed. In vivo experience in rodent models, such as dosing, tissue collection, necropsy/dissection, and behavioral or functional phenotyping, is also required.

Preferred Qualifications

• Experience with AAV biology and workflows, including vector titering.
• Strong mathematical skills are preferred.
• A collaborative, team-oriented working style paired with the independent judgment to drive projects forward proactively in a fast-paced environment.
• Strong scientific communication skills, with the ability to synthesize data for both technical and cross-functional audiences.