Description
We are seeking a highly skilled and experienced Scientific Investigator to join our team of scientists with the JAX Rare Disease Translational Center (RDTC). This role will be the lead technical scientist in gene editing therapeutics design and development. The successful candidate will provide technical design and scientific expertise, work with external manufacturing organizations, lead an independent research program in rare disease therapeutic development, and drive translational science to bring new therapeutics to the clinic. Reporting to the VP, RDTC, this role requires a deep understanding of generating and testing gene editing strategies and therapeutics for preclinical efficacy and pharmacology studies, clinical trials and commercialization.
The JAX RDTC leverages JAX’s unique research environment and resources to translate biological discovery into clinical action. We work directly with foundations and their associated teams of basic science and clinical researchers to understand the molecular underpinnings of disease symptoms, identify pathways that can be targeted therapeutically, and assess the safety and efficacy of candidate drugs and therapies in biologically accurate mouse models of disease. This position is an exciting opportunity to work directly with the RDTC in supporting this mission.
Key Responsibilities:
Lead technical design and provide scientific expertise for gene editing technology and therapeutics.
Establish in vitro and in vivo gene editing screening and validation protocols in cellular and animal models.
Perform manufacturing vendor evaluations and oversee outsourced work.
Lead an independent research program in rare disease therapeutic development, under the direction of RDTC leadership.
Work as part of cross-functional teams including peer scientists, patients, advocacy groups, commercial clients, clinical partners and regulatory agencies to evaluate, prioritize, validate and advance therapeutics projects to the clinic.
Collaborate with RDTC leadership, lab scientists and data scientists to develop and advance partnership and commercialization strategies.
Stay abreast of emerging technologies and regulatory landscapes in the field.
Lead gene- based therapy R&D projects to learn and advance the technology and assets.
Develop and oversee quality and process control adherence to technical specifications for gene editing.
Troubleshoot technical issues and devise solutions.
Serve as a subject matter expert, submitter and superuser for the utilization, development, expansion and integration of relevant oligonucleotide database platforms and repositories.
Contribute to or lead grants.
Contribute to or lead collaborative research projects and publications.
Communicate effectively to manage internal and external relationships.
Requirements
Requirements:
PhD in Chemistry, Biochemistry, Chemical Engineering, Biology or a related discipline, with 5+ years of experience beyond the doctoral degree.
Extensive experience in gene editing science, methods, strategies and therapeutics.
Proven abilities in data-driven decision-making and problem-solving.
Strong knowledge of quality process control and vendor quality assessments.
Ability to lead independent research initiatives, analyze experimental data, and interpret results.
Experience working with cross-functional teams in the development and use of databases.
Experience in securing and managing research grants and budgets.
Strong mentorship skills to guide graduate students, postdoctoral researchers, and other team members.
Ability to collaborate with interdisciplinary teams and external partners.
Understanding of regulatory pathways and compliance.
Creative problem-solving skills in research and experimental design.
Commitment to ethical research practices and adherence to safety protocols.
Focus on rare diseases and translational research to improve human health.
Effective budget management and laboratory maintenance skills.
Excellent communication skills for managing collaborations and presenting findings.
