Cell and Gene Therapies

We provide comprehensive support for the development of cell and gene therapies (CGTs), addressing the unique challenges posed by their complexity. From discovery to regulatory submission, our customized services and expertise ensure seamless progression through nonclinical development, regulatory approval, and clinical trials.

Our goal is to support you throughout your cell and gene therapy development journey by offering customized solutions that accelerate R&D efforts or generate IND-enabling studies. With expertise in assay development, biomarker discovery, CMC strategies, and safety testing, we provide the tools you need to advance your therapies confidently from discovery to clinical application.

• Consultation and Planning: Strategic guidance tailored to streamline CGT development from discovery to clinical trials
• Nonclinical Support: Comprehensive studies to evaluate efficacy, safety, and translational potential of CGT modalities
• Assay Development and Validation: Creation and optimization of robust assays to assess potency, specificity, and functionality
• CMC Strategies: Customized solutions for characterization and regulatory compliance of CGT products
• Safety Testing: Advanced assays to predict adverse immune-related effects, neurotoxicity, and other safety risks
• Data Analysis and Reporting: High-quality data interpretation with actionable insights for regulatory submissions
  

Accelerating ATMPs from discovery to clinical trials

• We specialize in delivering Advanced Therapy Medicinal Products (ATMPs) from early proof-of-concept to first-in-human (FIH) clinical trials. We deliver robust nonclinical data packages, essential for regulatory approval and long-term success, by designing IND-enabling studies and leveraging advanced tools such as:Multiplex Luminex
• MSD, flow cytometry
• Next-generation sequencing (NGS),
• In vivo/in vitro disease models
• Spatial biology,

Expertise Across CGT Modalities

Our extensive capabilities span diverse cell and gene therapy approaches:

• Cell therapies: Stem cells (iPSC, MSC), macrophages, tumor-infiltrating lymphocytes (TILs), regulatory T-cells (Tregs), CAR-T, CAR-M, CAR-NK, TCR-T therapies.

• Gene therapies: Oncolytic viruses, adeno-associated viruses (AAVs), lentiviral vectors (LVVs), lipid nanoparticles (LNPs), virus-like particles (VLPs), nanoparticles, extracellular vesicles (EVs), mRNA vaccines, RNAi therapeutics.

We ensure robust and predictive evaluations using tailored models that closely mimic disease conditions.

Advanced efficacy and toxicity evaluation

We offer state-of-the-art cell-based assays for safety, specificity, and efficacy assessments across therapeutic areas such as:

• Oncology
• Immuno-oncology
• Neuroscience
• Immunology
• Inflammation
• Autoimmunity

Our services include:

• Cell kill assays: Evaluating tumour-killing efficacy in 2D and 3D models
• On/Off-target assays: Ensuring safety through off-target binding detection
• Mixed lymphocyte reaction (MLR): Evaluate immunotoxicity risks like Graft-versus-Host Disease (GvHD)
• Exhausted T cell assays: Measuring markers of immune cell exhaustion to ensure sustained functionality
• Cytokine release assays (CRA): Quantify cytokine levels to evaluate risks of Cytokine Release Syndrome (CRS) and immune-mediated toxicity
• Pro-inflammatory cytokine profiling: Modelling inflammatory responses to manage adverse immune-related effects effectively
• Neurotoxicity profiling:  Developing predictive assays to evaluate risks such as Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS)

These comprehensive platforms ensure the development of safer therapies with confidence.

Analytical development

CGTs present unique challenges in chemistry, manufacturing, and controls (CMC). We provide customized analytical services for extensive characterization:

• Physicochemical profiling and stability assessments
• Interaction analysis & structural biology for affinity and kinetics evaluation
• Payload encapsulation efficiency for RNA/DNA/protein delivery systems
• Transfection efficiency analysis in cellular models

Our orthogonal analytical techniques ensure robust characterization of complex gene therapy products.

Biomarker development and exploratory analysis

We support biomarker discovery, validation, and companion diagnostic (CDx) development using advanced platforms like spatial biology and multiplex assays.

Our expertise enables:

• Identification of predictive or prognostic biomarkers
• Patient stratification for optimized therapeutic outcomes
• Mechanism-of-action studies to refine clinical trial designs

By integrating exploratory endpoints with biomarker insights, we help you optimize therapeutic strategies effectively

Target discovery and validation with spatial biology

Our spatial biology services provide precise insights into biomarker distribution and cellular interactions within tissues. Using advanced multiplexed imaging and spatial transcriptomics, we identify key therapeutic targets within their native microenvironment.

Complemented by in vitro models such as 2D/3D cultures and flow cytometry platforms, we deliver robust target validation for CAR-T therapies, TCR-based approaches, gene-editing strategies, and beyond.

Our experienced team is happy to discuss your CGT project at any stage.

Book a meeting with an expert today to disucss your specific project needs.