Role Summary
Scientist with deep expertise in in vitro assay development to support expanding therapeutic programs. This hands‑on, lab‑based role is responsible for designing, developing, and executing cellular assays to evaluate exosome‑based delivery of therapeutic agents, including protein, mRNA, siRNA, ASO, and PMO. The ideal candidate will have strong technical proficiency, problem‑solving skills, and a passion for advancing novel therapeutic platforms. Collaboration across molecular biology, cell biology, and translational research teams is required, and experience with iPSC genome editing and differentiation into myogenic lineages is highly desirable. This on‑site role is based in Capricor’s San Diego research facilities.
Responsibilities
- Design, develop, and validate mechanism‑of‑action and functional in vitro assays to assess therapeutic efficacy, potency, and safety
- Establish PK/PD assay platforms to characterize cellular uptake, expression, and downstream biological responses
- Utilize a range of assay technologies including high‑content imaging, flow cytometry, qPCR, Western blotting, and ELISA
- Develop and optimize exosome or lipid nanoparticle loading and delivery protocols for protein, mRNA, siRNA, ASO, and PMO payloads
- Characterize loading efficiency, payload integrity, delivery kinetics, and functional outcomes in vitro
- Culture and manipulate diverse mammalian cells including primary cells, immortalized lines, and iPSCs
- Perform transfection, transduction, and nucleic acid delivery followed by molecular and functional analyses
- Support CRISPR/Cas9 or related gene editing workflows
- Develop and apply disease‑relevant in vitro models for rare muscle disorders, including iPSC‑derived myoblasts
- Correlate cellular phenotypes with disease‑relevant biomarkers and therapeutic mechanisms
- Troubleshoot and optimize complex assay workflows and delivery methodologies
- Analyze and interpret data; prepare clear presentations and written reports
- Maintain rigorous documentation and data integrity standards
- Work collaboratively with research, bioanalytics, and translational teams to align assay strategies with program objectives
- Stay current with emerging technologies in drug delivery, nucleic acid therapeutics, assay platforms, and exosome science and apply innovative thinking to accelerate discovery
Qualifications
- Ph.D. in Cell Biology, Molecular Biology, Biochemistry, or a related field with 0–3+ years of postdoctoral or industry experience; or M.S. with 5+ years of relevant experience
- Proven hands‑on experience in in vitro assay design, development, and validation for therapeutic evaluation
- Proficiency with mammalian cell culture, including primary cells, immortalized lines, and (ideally) iPSCs
- Experience with exosome‑based or related drug substance delivery platforms (e.g., lipid nanoparticles) for protein, mRNA, siRNA, ASO, or PMO payloads
- Technical capability with high‑content imaging, flow cytometry, qPCR, Western blotting, ELISA, and related analytical methods
- Experience performing transfection, transduction, nucleic acid delivery, and subsequent molecular/functional analyses
- Understanding of CRISPR/Cas9 genome engineering and, ideally, iPSC genome editing and differentiation into myogenic lineages such as myoblasts
- Familiarity with rare muscle disease biology, associated cellular phenotypes, and relevant biomarkers
- Working knowledge of nucleic acid and protein‑based therapeutic modalities including siRNA, ASO, PMO, mRNA, and proteins
- Strong analytical skills with proficiency in data analysis tools such as GraphPad Prism and FlowJo
- Demonstrated ability to troubleshoot complex biological systems and optimize experimental workflows
- Highly organized with strong attention to detail and excellent documentation practices
- Effective verbal and written communication skills; proven ability to work collaboratively across cross‑functional teams
Education
- Ph.D. in Cell Biology, Molecular Biology, Biochemistry, or related field with 0–3+ years of postdoctoral or industry experience; or M.S. with 5+ years of relevant experience
Skills
- In vitro assay design, development, and validation
- Mammalian cell culture (primary cells, immortalized lines, iPSCs)
- Exosome‑based or lipid nanoparticle delivery platforms
- High‑content imaging, flow cytometry, qPCR, Western blotting, ELISA
- Nucleic acid delivery (transfection/transduction) and downstream analyses
- CRISPR/Cas9 genome engineering; iPSC differentiation to myogenic lineages
- Data analysis (GraphPad Prism, FlowJo); strong problem‑solving and documentation
Additional Requirements
- On‑site laboratory role based in San Diego
- Regular bench work; occasional lifting of up to 25 lbs
- Adherence to biosafety practices and documentation procedures