Gene Therapies

Bespoke scientific expertise to expedite your gene therapy program

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

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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.

  • Discovery/Preclinical

    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
  • GMP CMC Release

    Critical quality attributes

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

    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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