Senior Rare Disease Therapeutics Research Scientist

6 days ago

Bar Harbor, Maine, United States The Jackson Laboratory Full time

The Jackson Laboratory is a renowned biomedical research institution with a National Cancer Institute-designated Cancer Center and over 2,900 employees in locations across the United States, Japan, and China. Our mission is to uncover precise genomic solutions for disease and empower the global biomedical community in its quest to improve human health.

We are seeking an experienced Senior Rare Disease Therapeutics Research Scientist to join our team of scientists at the JAX Rare Disease Translational Center (RDTC). This role will be the lead technical scientist in nucleic acid therapeutics design and development, working closely with external manufacturing organizations, leading independent research initiatives, analyzing experimental data, and interpreting results.

The successful candidate will have a deep understanding of generating and testing oligonucleotide therapeutics for preclinical efficacy and pharmacology studies, clinical trials, and commercialization. They will also have experience working with cross-functional teams in the development and use of databases, securing and managing research grants and budgets, and collaborating with interdisciplinary teams and external partners.

This position offers an exciting opportunity to work directly with the RDTC in supporting our mission to translate biological discovery into clinical action. We leverage JAX's unique research environment and resources 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.

Key Responsibilities:
  • Lead technical design and provide scientific expertise for oligonucleotide therapeutics
  • Establish in vitro and in vivo ASO 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 ASO products
  • 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

We offer a competitive salary range of $111,488 - $186,656 based on experience.

Requirements:
  • PhD in Chemistry, Biochemistry, Chemical Engineering, Biology, or a related discipline, with 5+ years of experience beyond the doctoral degree
  • Extensive experience in oligonucleotide or synthetic peptide design
  • 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

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