Gene Therapy Stages of Development
When developing gene therapies from concept to medicine, we understand that your top priority is to accelerate your journey to market, enabled by in-depth insights and accurate decision-making. With advanced analytical solutions, we can provide you with the support you need to fast-track your development while ensuring full compliance with rigorous regulatory standards.
Our wide range of high-quality reagents and analytical instruments are designed to streamline every step of your journey, from serotype identification to ensuring clinical success. Swiftly bring your gene therapy to market without compromising quality, safety, or efficacy.
Target and Lead Discovery
Discovering novel gene candidates and lead constructs, while evaluating their suitability for gene therapy interventions can be extremely time-consuming and complicated. Our high-throughput, automated solutions enable you to evaluate potential targets and vectors quickly and accurately, accelerating your journey to discover safer and more effective therapeutic interventions.
Featured Solutions
Isoform Analysis
Isoform Analysis
Characterization of adeno-associated virus (AAV) and lipid nanoparticle (LNP) delivery vectors is key to ensuring the safety and efficacy of your therapy. Maurice makes measuring the surface charge of these large, complex particles easy while saving you time, sample, and labor.
Protein Expression Screening
Protein Expression Screening
Simple Western platforms enable high-throughput, quantitative protein expression characterization directly in cell and tissue lysates. Quickly evaluate target gene candidates, delivery vehicles, and transgene expression at the protein level to select your gene therapy candidates with confidence.
Fast Functional Characterization
Fast Functional Characterization
The Ella™ automated immunoassay platform offers a menu of validated ready-to-use assay kits for easy measurement of secreted cytokine protein expression in cell culture supernatant. Quickly evaluate functional characterization in less than 90 minutes.
Preclinical Gene Therapy Testing
During preclinical testing, therapeutic candidates are evaluated for efficacy, potency, and viral titer, both in ex and in vivo using suitable disease models. Our range of customizable and automated analytics ensures that promising therapeutics will progress smoothly.
Featured Solutions
Rapid Characterization
Rapid Characterization
Ella™ is a benchtop, automated immunoassay platform that delivers rapid and precise measurement to boost development and manufacturing efficiency. Get high-quality, quantitative data in less than 90 minutes with unparalleled flexibility and ease of use.
Do More With Less
Do More With Less
Simple Western capillary immunoassay platforms offer reproducible, quantitative potency measurements directly in cell and tissue lysates. With just 3 µL of sample, platforms like Jess™ and Abby™ also allow you to characterize additonal critical quality attributes, including empty-full and capsid protein ratios.
Toxicity Testing
How can you determine if your therapeutic is performing appropriately in your disease model? Through accurate and reliable testing that can assess cytokine profiles, AAV purity, and the biodistribution of your product. With extremely sensitive assays, we can ensure that your product is working exactly as intended.
Featured Solutions
Sensitive and Easy
Sensitive and Easy
The Ella platform provides high sensitivity and large dynamic range to quantify process-related and host-cell related impurities in less than 90 minutes. Ready-to-use assay kits are available for Endonuclease, HEK, CHO, and more.
Sub-Cellular Resolution
Sub-Cellular Resolution
RNAscope technology visualizes AAV vector and transgene biodistribution in tissue, combining quantitative molecular measurement with single-cell resolution. RNAscope is a leading FDA-recommended choice for AAV biodistribution, enhancing therapeutic efficacy and safety assessment.
Gene Therapy Clinical Testing
Clinical testing, a resource-intensive and time-consuming stage in the path to commercialization, requires analytical tools that are easy to use, reliable, and accurate for widespread use and comparison. Implementing a comprehensive analytical approach to evaluating therapeutics ensures that new treatments are not only safe and effective but also benchmarked against existing standards, ultimately leading to better patient care.
Featured Solutions
Precise Analysis
Precise Analysis
The Maurice system combines icIEF and CE-SDS analysis for comprehensive data in one compact instrument. Get automated identity, purity, and stability data with low sample volumes in as little as 5.5 minutes for CE-SDS or 10 minutes for icIEF. This automated method offers enhanced precision, reproducibility, and throughput, making it ideal for viral vector analysis.
Easy, Validated Assays
Easy, Validated Assays
Ella™ delivers sensitive detection and broad dynamic range throughout clinical testing. With a four-log range of detection, Simple Plex assays have the sensitivity to pick up even very low picogram-per-milliliter levels of endogenous analytes.
Small Sample Efficiency
Small Sample Efficiency
Simple Western capillary immunoassay platforms offer reproducible, quantitative potency measurements directly in tissue lysates. Using only 3 μL of sample per capillary, platforms like Jess™ and Abby™ allow you to conserve precious clinical samples, while maximizing data output with high sensitivity detection, more than one round of immunoprobing, and total protein normalization.
For Diverse Conditions
For Diverse Conditions
Our Micro-Flow Imaging (MFI) systems directly image subvisible particles in solution to provide data for 10 different morphological parameters with precision and accuracy. The MFI can distinguish and classify different particle types, allowing you to identify AAV aggregates as well as any contaminants or impurities, ensuring the stability and safety of your therapeutic.
Analytics in Gene Therapy Development
As the field of gene therapy continues to evolve and grow, comprehensive analytical techniques are essential to ensuring all the critical quality attributes (CQAs) of your vector are met, optimizing your products for maximum therapeutic impact.
Vector characterization and in vivo monitoring are crucial aspects to any gene therapy development. Our suite of scalable and automated analytical solutions not only enhance the development of your gene therapy treatments but also prioritize patient well-being through meticulous, data-driven approaches.
Viral Vector Characterization
Comprehensive viral vector characterization is essential for optimizing processes, ensuring safety, and maximizing efficacy in gene therapy development. However, meeting stringent regulatory standards is highly challenging due to process variability, complex materials, and the ever-changing industry landscape. Using our expertise, you can make informed decisions supported by thorough analytical insights, accelerating your time to commercialization without compromising quality, safety, and efficacy. Our customizable, flexible, and automated assay and analytical systems can establish a streamlined and cost-effective workflow, allowing your therapeutics to reach the market faster to meet patient needs.
Utilizing our solutions, you can comprehensively characterize a multitude of AAV and lentiviral vector Critical Quality Attributes (CQAs), including:
- Titer: Quantify the amount of AAV titer
- Identity: Confirm serotype identity and verify capsid assembly for regulatory compliance
- Potency: Ensure sensitivity and reproducibility in verifying viral vector protein expression and transgene activity
- Purity: Overcome limitations related to sample volumes and turnaround times in analyzing genetic load and impurities
- Stability: Enhance long-term stability and efficacy by detecting aggregation and particle formation, minimizing long-term losses in efficacy and safety
- Biodistribution: Evaluate dosing, safety, and efficacy of your therapeutic through visualization of vector DNA and transgene mRNA expression in tissue
In Vivo Monitoring
Gene therapy still faces fundamental challenges, including potential immune responses, off-target effects, and abnormal transgene expression levels. Therefore, it is essential to assess therapy function at the target site and explore off-target effects, immune response, expression levels, and potential delivery into unintended cells or tissues.
Featured Solutions
Visualize AAV Biodistribution and Perform Safety Analysis
The high specificity and single-molecule sensitivity of the RNAscope technology makes it a key tool for the stringent assessment of gene expression in the tissue context. Evaluate the performance of your engineered cargo in vivo. FDA strongly recommends biodistribution studies to characterize AAV biodistribution and RNAscope ISH assay is the most powerful spatial method available within the methods provided. RNAscope helps characterize tissue biodistribution, cellular tropism, capsid selection and transduction efficiency of your vector.
Discover Powerful RNA Visualization with RNAscope at ACD »
High-Performance Detection
High-Performance Detection
Using our broad range of antibodies, antibody panels, and fluorokines, flow cytometry and immunohistochemistry (IHC) can be used to identify cell defects, profile the transgene, and for cellular spatial analysis.
High-Throughput Multiplexing
High-Throughput Multiplexing
Our Luminex assays maximize multiplexing capacity and flexibility while maintaining assay specificity. These bead-based assays ensure robust and reproducible data, rapidly quantifying immune responses and cytokine release. Customize panels using the extensive mix-and-match menu, with up to 50 targets analyzed per sample.
