Bespoke scientific expertise to expedite your gene therapy program

Pharmacodynamic response/efficacy/research and development of novel delivery modalities

• Longitudinal, in vivo imaging in rodents (IVIS)In-life, in vivo imaging in rodents (IVIS)
  
• Variety of targeted surgical techniques, including MRI-guided stereotaxic surgery
• IHC, ELISA
• Gene expression by RT-qPCR, NanoString

Toxicology and safety assessment

• GLP compliant in small- and large-animal models
• GLP formulation, dose analysis and administration, including delivery device compatibility testing
• Biodistribution and persistence qPCR or ddPCR, RT-qPCR, IHC, ELISA, neutralizing antibody (nAb), total antibody (tAb)
• Histopathology and IHC; board-certified clinical and anatomic pathologists
• Cytokine/chemokine analysis: multiplexed immunoassays
• Clinical pathology (e.g., clinical chemistry, hematology, urinalysis)
• Toxicology assessment for the projected Phase I doses

Critical quality attributes

• Genomic and capsid titer
• Potency
• Safety and replication competent vector
• Purity and impurities
• Identity
• Stability

Patient selection/predictive biomarkers

• Clinical biomarkers (Luminex, MSD, Quanterix, ELISA)
• Preexisting immunity by ELISA, cell-based assays, companion diagnostics
• Genomics (including NGS)

Safety

• Shedding by PCR
• Circulating biomarkers (using Luminex, MSD, Quanterix, ELISA, etc.)
• Immunogenicity: ELISpot analysis to vector and transgene
• ADA to vector and transgene
• Vector site integration

Target identification and patient selection/predictive biomarkers

• Gene expression (NanoString, NGS, PCR, single-cell sequencing)
• Multiplex cytokine and chemokine assays (MSD, Luminex, Quanterix)
• IHC
• Flow cytometry