Scientist/Sr. Scientist, Drug Discovery

Job Title: Scientist/Sr. Scientist, Drug Discovery

Job Number: 33268

Location: , CA

Job Description

Position Summary: We are currently recruiting a highly skilled and independent Scientist/Senior Scientist to join our team to help support our discovery research efforts. This laboratory-based position will be responsible for progressing our pipeline by independently designing, executing, and optimizing experiments involving novel gene therapies for neuromuscular disease indications. This position will require creativity, flexibility, and a solid understanding of molecular biology and drug development for testing novel therapeutic approaches. The candidate will also need to develop genetic screens to optimize gene therapy regulatory elements to enable pharmacological assessment in mouse models of disease. This position will collaborate with other scientists, including computational biologists, in vivo pharmacologists, process development scientists, and other external scientists across academia and industry to advance the pipeline from idea to clinical development. As we are a start-up company, this position has tremendous room for growth and opportunities across multiple areas of R&D.

Responsibilities

Key Responsibilities:

• Design, construct, and optimize candidate gene therapies in vitro using a variety of cell-based models
• Translate in vitro findings to mouse models of disease in collaboration with pharmacology, by establishing disease models, and molecular biomarkers of pharmacology and other endpoints to assess efficacy and safety-based endpoints
• Collaborate closely with other discovery scientists to understand program needs and timelines
• Execute a range of cellular and molecular biology workflows to support program progression across pipeline
• Integrate into matrixed project teams and coordinate resources to ensure completion of milestones
• Contribute expertise in gene therapy and/or neuromuscular disease biology to generate high-quality data
• Identify opportunities to improve existing protocols or establish new assays as needed
• Prepare summaries of data and present internally to colleagues and management
• Draft SOPs, follow protocols, and diligently document experimental data in electronic lab notebooks
• Actively support a culture of innovation, learning, respect, and trust

Required Skills

Desired Qualifications

• PhD and postdoctoral training in biological sciences, with an emphasis on muscle disease biology, genetic engineering, molecular and cellular biology and/or related fields. Prior industry experience is a plus. Title will be commensurate with experience.
• Proficiency in vector design, cloning, transfection, cell culture, cell imaging, nucleic acid sequencing, and qPCR/ddPCR techniques. Direct experience with AAV viral gene therapy in preclinical research is preferred.
• Proven experience in developing cell-based models using a variety of molecular, biochemical, immunological, and/or imaging-based endpoints, preferably in the neuromuscular disease field.
• A record of productivity and scientific excellence based on consistent high-impact publications, patents, and/or external presentations.
• Independent and critical thinker, multi-tasker, and problem solver with strong verbal and written communication skills, and a team-first mindset.