Our client using the emerging tools of genetic medicine to engineer cures for human diseases. We believe these powerful new approaches can be used to treat, or cure, common diseases that afflict large numbers of patients. To realize this vision, we are building therapeutics that modify and control multiple genes at the DNA, RNA, and protein level in vivo. We have a vibrant, collaborative workplace that supports our team in performing great science while bringing their whole selves to work. We are seeking several highly motivated, creative individuals to join our growing team.

About the Role:

As a Principal Scientist reporting into the Head of Preclinical, you design, execute, and analyze experiments from therapeutic target evaluation to developmental candidate nomination. This position will be leading from the bench, and you will be working in close collaboration with our therapeutic program and platform technology teams, you will:

• Execute experiments to identify lead candidates and optimize them to improve efficacy and safety of the platform.

• Demonstrate therapeutic proof-of-concept, including identifying and onboarding disease-specific in vivo models.

• Develop in vivo delivery approaches for candidate therapies using AAV, LNP, or other platforms.

• Coordinate assay development and execution of bioanalytical assays and other study read-outs, working with both internal and external resources.

• Manage, oversee, train, and mentor scientists and research associates and foster a collaborative e environment within the team and with external partners.

• In all cases, you will ensure that study design and properly documented and align with the company's research goals, timelines, and budgets.

Requirements:

• A Ph.D. in neuroscience, pharmacology, physiology, drug delivery, genetics or a related life sciences discipline, with at least five years of drug development experience.

• In-depth knowledge of animal models used in drug development research and experience in applying these models to in vivo studies.

• In depth knowledge of iPSC-derived models in drug development and experience in applying these models to in vitro studies.

• Strong leadership abilities, with experience in managing scientific teams and fostering collaborative internal and external research environments. Demonstrated ability to work closely with senior leadership to ensure research efforts align with organizational strategy.

• Ability to think creatively and drive for success in a fast paced and agile work environment.

Preferred Qualifications:

• Experience using CRISPR-Cas or similar gene editing tools for therapeutic applications

• Demonstrated ability to work in multi-disciplinary teams to achieve experimental objectives

• Experience working with gene editing and/or gene therapy applications, including refinement of core technology for specific targets.

• Experience working in dynamic startup and/or small company environments that necessitate pitching in and assisting with a wide variety of important tasks.