With rapid advances in genetic engineering technologies, gene therapy offers groundbreaking potential to treat a wide range of genetic disorders. At Scantox, we provide scientific guidance and comprehensive preclinical services to take these innovative discoveries from concept to IND-enabling studies.
Integrated Service Solutions
Understanding the complexities of gene therapy modalities requires robust in vitro and in vivo models. At Scantox, we use these models to identify and validate therapeutic targets and evaluate their safety. Our capabilities extend to conducting animal studies across various biosafety levels, including BSL-1, BSL-2, and even BSL-3.
Our extensive expertise in non-viral and viral gene therapy candidates (AAV, adenoviruses, oncolytic viruses, etc.), coupled with a highly skilled team specializing in molecular, histological, and biochemical analyses, ensures that each study we undertake is supported by accurate evaluations and reliable data.
In vitro Gene Therapy Services
To optimize your gene therapy strategies, we offer customized viral transduction protocols across a diverse range of cell systems, including:
- Various neuronal- and non-neuronal cell lines (e.g. SH-SY5Y, HEK293T, HepG2…)
- Primary mouse and rat cells
- Mouse embryonic fibroblasts
- Human fibroblasts (diseased and healthy controls)
- Human induced pluripotent stem cells (iPSCs)
- Organoids (in the pipeline)
Key in vitro readouts include:
- Viability and cytotoxicity assessments
- Life cell imaging
- Neuronal activity monitoring
- Tests for oxidative stress and mitochondrial activity
- Comprehensive efficacy testing
These in vitro capabilities form the foundation for in vivo applications, ensuring a seamless transition from lab bench to animal model.
In vivo Gene Therapy Services
In vivo services rely on advanced animal models and precise surgical techniques to administer gene therapy agents through various methods, ranging from parenteral to stereotactic injections. We focus on two key objectives: maximizing efficacy and minimizing invasiveness. Our capabilities also encompass administering gene therapy agents to larger animals like rabbits and minipigs.
Key in vivo readouts include:
- Pharmacokinetics and pharmacodynamics (PK/PD) and biodistribution
- Safety and general toxicology evaluations
- Efficacy studies and clinical monitoring
Capabilities of gene therapy application routes across preclinical models:
Sample collection capabilities:
We enable a longitudinal assessment of biomarkers by repeatedly sampling high-quality biofluids and tissues for ex vivo analyses.
Biofluids:
- Blood
- Cerebrospinal fluid (CSF)
- Bronchoalveolar lavage
Tissues:
- Whole brain or individual brain regions
- Spinal cord
- Dorsal root ganglia
- Peripheral organs
- Various muscles
Technological Assets for Precise Evaluations
We provide integrated technological solutions to accurately track target gene and protein expression and therapeutic efficacy ex vivo and in vivo:
- qRT-PCR: to quantify the presence and distribution of transgenic material within target tissues.
- Fluorescence In Situ Hybridization (FISH): to detect transgenic vectors and provide spatial information on vector localization
- In vivo imaging with the IVIS-CT system: to enable real-time non-invasive monitoring of specific reporter gene expression (e.g. luciferase)
- Western Blot/Automated Western Blot (WES): to detect specific proteins
- Standard ELISA & Multiplex ELISA (MSD platform): to measure detailed biomarker concentrations
- Immunohistology: for customized sectioning and staining for detailed tissue analysis
- Magnetic-Activated Cell Sorting (MACS): to isolate specific cell subpopulations, such as microglia
Why Choose Scantox?
- Extensive experience: Over 100 completed gene therapy studies using both in vitro and in vivo methods, including cell therapies, adenoviruses (AAVs), lentiviruses (LVs), antisense oligonucleotides (ASOs), and RNA therapeutics such as siRNA.
- Therapeutic area expertise: Highly trained scientific staff with specialized knowledge in CNS/neurology, rare diseases, oncology, metabolic diseases, infectious diseases, and immunology.
- Customization: Unique in vivo models and tailored routes of administration and technological assets to meet specific research needs.
- Commitment to excellence: Known for our transparency and reputation for delivering reliable, high-quality data
Engage with our specialized scientific experts to explore how we can support your research and development goals.