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Bespoke scientific expertise to expedite your gene therapy program

Gene therapies

Unique in vivo models, complex methods and routes of administration, to streamline your development

  • Therapeutic area expertise for indications including: neurology, cardiovascular disease, ophthalmology, immunology and more

  • Custom assay support for preclinical, biomarker-driven clinical trial testing and commercialization of your potential companion diagnostic

  • Robust global laboratory experience supporting of 7 FDA-approved gene replacement therapies


A partner for your gene therapy testing needs—wherever you are in development

Whether you’re developing a viral vector-based therapy, such as an adeno-associated virus or lentivirus product, or applying gene editing technologies such as CRISPR/cas9, we’re here to offer you the type of support you need—when you need it—so you can reach your development goals on time and within budget.

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

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