Research Associate II/ Senior Research Associate, In Vitro Pharmacology

Responsibilities

• Design, execute, analyze, and interpret in vitro pharmacology experiments that support therapeutic candidate discovery and advancement.
• Develop and optimize cellular assays to evaluate CRISPR-based epigenetic editing systems, including potency, durability, specificity, and mechanism of action, to inform program decisions.
• Generate high-quality datasets and apply rigorous analysis to support data-driven program decisions.
• Partner closely with cross-functional teams to advance therapeutic programs.
• Culture, engineer, and characterize mammalian cell lines, primary cells, and iPSC-derived disease models.
• Perform molecular and cellular analyses including qPCR, RT-qPCR, digital PCR, Western blotting, ELISA, flow cytometry, immunofluorescence, and next-generation sequencing-based assays.
• Evaluate delivery and activity of gene therapy modalities, including viral (AAV, Lenti) and non-viral delivery systems.
• Maintain detailed experimental records and contribute to study reports, presentations, and regulatory documentation.
• Stay current with emerging technologies and scientific advances in CRISPR, epigenetics, gene therapy, and neuromuscular disease research.

Requirements

• Research Associate II
• B.S. or M.S. in Molecular Biology, Cell Biology, Genetics, Neuroscience, Biomedical Engineering, or related field.
• 3-5+ years of relevant industry experience.
• Senior Research Associate
• B.S. or M.S. with 5-8+ years of industry experience.
• Demonstrated ability to independently execute complex studies and contribute to experimental strategy.
• Core Technical Skills:
• Hands-on experience with CRISPR technologies, including CRISPR-Cas9, CRISPRi, CRISPRa, or epigenome editing (Preferred) platforms.
• Strong molecular biology skills including nucleic acid extraction, PCR, qPCR, dPCR, cloning, and gene expression analysis.
• Mammalian cell culture and cellular assay development experience.
• Experience with viral vector systems (AAV, lentivirus, or related gene therapy platforms).
• Proficiency in flow cytometry, immunofluorescence staining, microscopy, and protein expression analyses.
• Strong data analysis, experimental design, and troubleshooting skills.
• Experience in epigenetics, chromatin biology, transcriptional regulation, or gene expression control.
• Experience working with neuromuscular diseases such as Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), FSHD, myotonic dystrophy, ALS, SMA, or related disorders.
• Experience with myoblasts, human iPSC-derived skeletal muscle or other disease-relevant cellular models.
• Familiarity with transcriptomics, NGS, ATAC-seq, ChIP-seq, or other genomic profiling technologies.
• Prior experience supporting preclinical therapeutic development programs in biotechnology or pharmaceutical settings.
• Knowledge of translational biomarkers and pharmacology assay development.
• What Will Make You Successful:
• Scientific curiosity and a strong desire to understand biological mechanisms.
• Ability to think critically about experimental design and data interpretation.
• Ownership mindset with attention to detail and commitment to high-quality science.

Benefits

Additional Information

Who Are We? Our company is based on the science of our founder, Stanley Qi, one of the original CRISPR co-inventors who then furthered the technology so that DNA does not need to be cut to accomplish gene regulation. Instead, we regulate the epigenome to suppress and activate multiple genes simultaneously. We are further evolving the platform and leveraging its strengths to address unmet medical needs. We are looking for exceptional team members who want an active role in building a rapidly growing biotech. Title: Research Associate II/ Senior Research Associate, In Vitro Pharmacology Location: South San Francisco, CA Department: Therapeutics Reports To: Principal Scientist, Pharmacology and Bioanalytical Development Opportunity: We are seeking an exceptional Research Associate II or Senior Research Associate to join our In Vitro Pharmacology team. In this role, you will help advance CRISPR-based epigenetic therapies from discovery through preclinical development, with a focus on neuromuscular and rare genetic diseases. This position is ideal for a highly motivated scientist who enjoys hands-on experimentation, critical data analysis, and scientific problem-solving in a fast-paced biotechnology environment, with the opportunity to take increasing ownership of projects and grow into more independent scientific roles. Position Summary: In this role, you will design and execute in vitro studies to characterize the activity, potency, mechanism of action (MoA), and translational potential of CRISPR-based gene regulation therapeutics. The ideal candidate brings hands-on expertise in molecular and cellular biology, gene editing technologies, and disease-relevant cellular models. Experience in neuromuscular disease biology, gene therapy, or epigenetic regulation is highly desirable.

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