Principal Scientist

About the role

Confidential Biotech | Stealth Mode | On-Site in MA We are partnering with a well-capitalized, stealth-stage biotechnology company developing a differentiated therapeutic platform with the potential to address significant unmet medical needs. We are seeking an accomplished and highly collaborative scientist to lead the Cellular Biology function.

Responsibilities

• Lead and build the Cellular Biology function, establishing scientific strategy, experimental capabilities, and rigorous standards for cellular and mechanistic biology.
• Develop and implement cell-based translational models of genetically defined human hematologic diseases, with particular emphasis on bone marrow and hematopoietic biology.
• Design and execute studies that characterize the pharmacodynamic and mechanistic properties of novel therapeutic molecules.
• Apply deep expertise in hematology and bone marrow biology to identify novel druggable targets, therapeutic approaches, and opportunities addressing unmet needs in priority hematologic diseases.
• Investigate kinase-driven and other intracellular signaling pathways to understand target biology, therapeutic response, and downstream cellular effects.
• Apply functional genomic and epigenomic approaches including scRNA-seq, ATAC-seq, and CUT&RUN to characterize cellular states, transcriptional programs, chromatin regulation, and therapeutic mechanisms.
• Integrate cellular, signaling, genomic, and epigenomic data to generate mechanistic hypotheses and guide drug-discovery strategy.
• Develop and optimize fit-for-purpose cellular and molecular assays, including flow cytometry, PCR, ELISA, and primary-cell culture.
• Generate data to support therapeutic candidate selection and prioritization and to inform go/no-go decisions.
• Help shape the future discovery and innovation portfolio through identification and evaluation of new biological hypotheses and therapeutic opportunities.
• Partner closely with cross-functional R&D stakeholders to ensure seamless study execution, timely data delivery, and alignment across the pipeline.
• Establish and manage CRO partnerships, external scientific collaborations, and internal capabilities supporting therapeutic identification, assay development, and candidate selection.
• Contribute to regulatory filings, scientific publications, data interpretation, and other activities supporting program and portfolio objectives.
• Mentor and develop scientists while fostering a culture of scientific rigor, innovation, accountability, and collaboration.
• Maintain substantial hands-on laboratory involvement during the initial build-out of the function, with bench work expected to represent approximately 50% of the role initially, decreasing as the team grows.

Requirements

• PhD with 8+ years of relevant experience in pharmacology, cell biology, hematology, molecular biology, or a related discipline; or MS with 10+ years of extensive relevant biopharmaceutical industry experience.
• Expert scientific and technical background in bone marrow biology and hematology, with demonstrated application to drug discovery, target identification, and/or development of novel therapeutic molecules.
• Extensive hands-on experience developing cell-based models of genetically defined human hematologic diseases.
• Strong expertise in kinase biology, phosphatase biology, and/or intracellular signaling pathways, with demonstrated experience using mechanistic studies to understand therapeutic response.
• Demonstrated experience with functional genomic and epigenomic approaches, including: single-cell RNA sequencing (scRNA-seq), ATAC-seq, CUT&RUN and/or closely related chromatin-profiling approaches.
• Ability to design rigorous genomic/epigenomic experiments, understand appropriate controls and technical considerations, critically interpret resulting datasets, and integrate findings with cellular and mechanistic biology.
• Deep hands-on expertise with core cellular biology techniques, including flow cytometry, PCR, ELISA, and cell culture.
• Demonstrated experience designing and implementing experimental strategies that generate mechanistic insight into hematologic disease biology and therapeutic correction.
• Proven scientific leadership, people leadership, and project management experience, including leading cross-functional, collaborative programs.
• Strong problem-solving, communication, organizational, and stakeholder-management skills.
• Preferred: Direct hands-on execution and troubleshooting of multiple functional genomic/epigenomic techniques, particularly scRNA-seq, ATAC-seq, and CUT&RUN.
• Preferred: Experience integrating multi-omic datasets with cellular phenotypes, signaling biology, and pharmacodynamic data.
• Preferred: Experience advancing therapeutic programs through candidate selection and toward IND-enabling development.
• Preferred: Experience contributing to regulatory filings.
• Preferred: Experience managing GLP- and/or cGMP-compliant workflows.
• Preferred: Experience establishing new laboratory capabilities and managing external CRO or academic collaborations.