Research Associate II/ Senior Research Associate, In Vitro Pharmacology

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.

Key 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.

Preferred Qualifications:

• 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