Scientist, In Vitro Assay Development & Drug Substance Delivery
18 days ago
San Diego
Job DescriptionCapricor Therapeutics (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapies for rare diseases. At the forefront of our innovation is Deramiocel (CAP-1002), our lead cell therapy in late-stage development for Duchenne muscular dystrophy. We are also harnessing our proprietary StealthX™ exosome platform to unlock new possibilities in targeted delivery and vaccinology. Every program reflects our commitment to pushing the boundaries of science and delivering life-changing treatments to patients and families who need them most. We are seeking a highly motivated Scientist deep expertise in in vitro assay development to support our expanding therapeutic programs. This hands‑on, lab-based role is critical in designing, developing, and executing cellular assays to evaluate exosome‑based delivery of therapeutic agents, including protein, mRNA, siRNA, ASO, and PMO. The ideal candidate brings strong technical proficiency, creative problem‑solving, and a passion for advancing novel therapeutic platforms. Experience with iPSC genome editing and differentiation into myogenic lineages is highly desirable. The role requires close collaboration across molecular biology, cell biology, and translational research teams to drive program success.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.Requirements, • 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.Work Environment, • This is a hands‑on, on‑site laboratory role based in Capricor’s San Diego research facilities., • Work is performed using standard cell‑culture and molecular‑biology laboratory practices, including routine use of biosafety cabinets and standard PPE., • Activities may involve human‑derived cell systems, gene‑delivery tools, and complex in vitro assays, requiring adherence to established safety and documentation procedures., • The position includes regular bench work, pipetting, computer‑based analysis, and occasional lifting of items up to ~25 lbs. Notice to Recruiting Agencies Capricor Therapeutics does not accept candidate submissions or referrals from recruiting agencies, staffing firms, or third-party recruiters without expressed consent from Talent Acquisition management and a prior written agreement. Agencies that contact hiring managers directly, solicit business, or submit candidates without this approval will not be considered for any engagement. All such submissions become the property of Capricor Therapeutics, and no fees will be paid for any candidates hired as a result. We appreciate your cooperation and respect for this policy.