Scientist, Nucleic Acid Delivery + In Vitro Assay Development

About Capricor Therapeutics

Capricor Therapeutics is a biotechnology company dedicated to developing transformative therapies for rare diseases, with a primary focus on Duchenne muscular dystrophy (DMD). We are pioneers in cell therapy and exosome-based technology, leveraging our proprietary StealthX platform to engineer innovative treatments. Our team is committed to harnessing cutting-edge science and advanced technologies to meet the unmet needs of patients living with DMD and other challenging medical conditions. Join us on our journey as we push the boundaries of medical science and pave the way for a healthier future.

Position Overview

We are seeking a highly motivated and skilled Scientist or Senior Scientist with strong expertise in in vitro assay development to support therapeutic programs focused on rare muscle diseases. The ideal candidate will play a critical role in designing and executing cellular assays to evaluate exosome-based delivery of therapeutic agents, including protein, mRNA, siRNA, ASO, and PMO. A demonstrated ability to troubleshoot complex biological systems and a passion for advancing innovative therapies are essential. Experience with iPSC genome editing and differentiation into myoblasts is a significant advantage. This hands-on, lab-based role involves strong cross-functional collaboration with molecular biology, cell biology, and translational research teams to advance Capricor's mission of developing transformative treatments for rare diseases.

Responsibilities

• In Vitro Assay Development:

• Design, develop, and validate mechanism-of-action and functional assays to evaluate the efficacy, potency, and safety of therapeutic candidates.

• Establish pharmacokinetic/pharmacodynamic (PK/PD) assay platforms to characterize cellular uptake, expression, and downstream effects.

• Employ advanced techniques such as high-content imaging, flow cytometry, qPCR, Western blot, and ELISA to support assay development.

• Exosome-Based Drug Substance Delivery:

• Develop and optimize loading and delivery protocols for therapeutic payloads (protein, mRNA, siRNA, ASO, PMO) into exosomes or lipid nanoparticles.

• Characterize loading efficiency, payload integrity, delivery kinetics, and functional outcomes in vitro using rigorous analytical methods.

• Cell Culture and Molecular Biology:

• Maintain and manipulate various mammalian cell types, including primary cells, immortalized lines, and preferably induced pluripotent stem cells (iPSCs).

• Perform nucleic acid delivery, transfection, and transduction, followed by molecular and functional analyses.

• Support genome engineering using CRISPR/Cas9 or related gene editing platforms.

• Disease Relevance:

• Develop and use in vitro models relevant to rare muscle diseases, including muscle-derived or iPSC-differentiated myoblasts.

• Correlate cellular phenotypes with disease-relevant biomarkers and therapeutic mechanisms.

• Troubleshooting and Optimization:

• Rapidly identify and solve technical challenges in assay workflows and delivery approaches.

• Adapt protocols to accommodate evolving project needs and new delivery modalities.

• Data Analysis and Reporting:

• Analyze and interpret complex data sets; communicate findings clearly through presentations and written reports.

• Maintain high standards of documentation and data integrity in a collaborative environment.

• Collaboration and Innovation:

• Work cross-functionally with research, bioanalytics, and translational teams to ensure alignment of assay strategies with therapeutic goals.

• Stay abreast of emerging technologies in drug delivery, nucleic acid therapeutics, and assay platforms, and apply innovative thinking to accelerate discovery.

Requirements

• Education: 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).

• Experience:

• Hands-on expertise in in vitro assay development and validation for drug evaluation.

• Hands-on expertise in exosome or similar delivery platform-based delivery of therapeutic agents.

• Hands-on expertise in mammalian cell culture, assay troubleshooting, and protocol optimization.

• Preferred Qualifications:

• Experience with iPSCs, including CRISPR-based genome editing and myogenic differentiation (e.g., into myoblasts).

• Familiarity with rare muscle disease models and relevant functional biomarkers.

• Knowledge of therapeutic modalities including siRNA, ASO, PMO, mRNA, and proteins.

• Proficiency in data analysis tools (e.g., GraphPad Prism, FlowJo).

• Soft Skills:

• Strong troubleshooting and problem-solving capabilities.

• Highly organized and detail-oriented with excellent documentation skills.

• Effective communicator and collaborative team player.

Work Environment and Physical Demands

• Professional laboratory setting with a focus on hands-on experimental work.

• Ability to perform laboratory tasks, including standing for extended periods, operating delicate equipment, and working in controlled lab environments.

• Ability to lift/carry up to 20 pounds and gown for classified cleanroom areas as needed for GMP compliance.

• Primarily an on-site role at our San Diego headquarters, with occasional flexibility for remote work based on project needs.

Compensation (USD)

Salary Range: $85,000 - $125,000 (commensurate with experience).

Benefits: Comprehensive benefits package, including health, dental, and vision insurance, retirement plans with company match, equity awards, generous paid time off, parental leave, and opportunities for professional development.

Why Capricor?

Capricor Therapeutics is an industry leader in cell and exosome therapy, pushing boundaries to create innovative treatments that raise the standard of care. Grounded in integrity and driven by science, our team is committed to making a meaningful impact through patient-centered innovation. Join us and be part of a team that's changing lives-help us shape the future of healthcare.

Come Work with Us!

At Capricor, we offer a collaborative work environment that supports your professional growth. Join a passionate team committed to transforming the lives of patients and pushing the boundaries of medical science. Apply today to contribute to our mission of developing transformative therapies for rare diseases like DMD.