mRNA Solutions
mRNA offers significant opportunities across a range of areas, including therapeutics, delivery systems, and in particular, vaccines. Our comprehensive preclinical testing services support mRNA programs from discovery through IND, enabling rapid development, scalability, and adaptability to emerging threats.
mRNA Solutions by WuXi AppTec Lab Testing Division
IND Enabling Packages
In Vitro Studies
In Vivo Studies
End-to-End Preclinical
Testing for mRNA
Our comprehensive services and capabilities cover every aspect of mRNA preclinical development, encompassing drug metabolism and pharmacokinetics (DMPK), bioanalytical, and pharmacology and toxicology testing—and all conveniently under one roof.
DMPK
Our global network of facilities, with locations in the US and China, offers a full range of discovery screening, preclinical development, and clinical drug metabolism and pharmacokinetic services.
Bioanalysis
We design bioanalytical strategies that allow us to identify and execute the right assays for regulatory submission, placing your mRNA candidate on the most promising path.
Safety Assessment
We assess mRNA toxicity by evaluating immunogenicity, off-target effects, local tissue reactions, systemic toxicity, genotoxicity, reproductive and developmental toxicity, and long-term safety.
CMC
We support your CMC authoring needs, in both the US and China, from preclinical to commercial lifecycle management for both drug substance and product.
Pharmacology
Get validated discovery assays, including in vitro assays and in vivo disease models in cardiovascular, CNS, respiratory, metabolic, and infectious diseases.
Regulatory Affairs
Make global filings convenient, efficient, and cost-effective across US FDA, NMPA, and EMA with our extensive regulatory submission support.
Comprehensive Preclinical Services
With capabilities in safety assessment, DMPK/ADME, bioanalytical, pharmacology, Chemistry, Manufacturing and Controls (CMC) and analytical services all under one roof, WuXi AppTec is a global expert in preclinical development testing and can support the full scope of your mRNA candidate, from discovery to IND-enabling studies to NDA submission.
Global Partner & Network
WuXi AppTec is a true global provider in preclinical small and large molecule solutions, with state-of-the-art facilities and experts across US, Europe and China. We have decades of experience operating within diverse regulatory environments and delivering tailored testing solutions that advance mRNA from preclinical through IND, NDA and beyond.
Regulatory Expertise
We guide you through the complexity of mRNA regulatory standards and guidance with technical expertise informed by our constant interaction with regulatory authorities. This knowledge is continuously incorporated and translated into actionable testing strategies that can accommodate strict requirements, ensuring smooth progression through regulatory approval.
Insights for mRNA Solutions
Frequently Asked Questions
What is mRNA and how is it used in therapeutics?
mRNA, or messenger RNA, is a type of RNA that carries genetic information from DNA to the cell’s protein-making machinery. In therapeutics, mRNA is used to instruct cells to produce specific proteins that can treat or prevent diseases, such as in mRNA vaccines or protein replacement therapies.
Why is preclinical testing important for mRNA-based therapies?
Preclinical testing is crucial for mRNA-based therapies to evaluate their safety, efficacy, stability, and delivery mechanisms before proceeding to clinical trials. It helps identify potential risks and optimize formulations to ensure effective and safe treatment.
What are the unique challenges in preclinical testing of mRNA compared to other types of therapeutics?
Unique challenges include ensuring mRNA stability, efficient delivery to target cells, avoiding degradation by nucleases, minimizing immune responses, and verifying that the encoded protein is correctly expressed and functional.
What types of studies are essential in the preclinical testing of mRNA?
Essential studies include in vitro assays for mRNA stability and protein expression, in vivo animal models for biodistribution, pharmacokinetics/pharmacodynamics (PK/PD), toxicology assessments, and immunogenicity evaluations.
How is the efficacy of mRNA therapeutics evaluated in preclinical studies?
Efficacy is evaluated by measuring the expression levels and activity of the encoded protein in target cells, assessing biological effects in disease models, and determining the therapeutic benefit in relevant animal models.
What safety assessments are critical for preclinical testing of mRNA therapies?
Critical safety assessments include toxicology studies to evaluate potential adverse effects, immunogenicity tests to assess immune responses, and biodistribution studies to determine the localization and persistence of mRNA in the body.
How are the pharmacokinetics and pharmacodynamics of mRNA studied in preclinical settings?
PK/PD studies involve measuring the absorption, distribution, metabolism, and excretion (ADME) of mRNA, as well as the time course of protein expression and its biological effects. These studies help determine the optimal dosing regimen.
What role do in vitro assays play in the preclinical testing of mRNA?
In vitro assays are crucial for initial screening of mRNA stability, delivery efficiency, protein expression, and functional activity. They help optimize mRNA sequences and delivery systems before moving to in vivo studies.
How are animal models used in the preclinical testing of mRNA therapies?
Animal models are used to study the in vivo efficacy, safety, biodistribution, and PK/PD profiles of mRNA therapies. These models help predict how mRNA treatments will behave in humans and identify potential therapeutic benefits and risks.
What are the regulatory considerations for preclinical testing of mRNA-based treatments?
Regulatory considerations include adhering to guidelines from agencies such as the FDA and EMA, conducting GLP-compliant toxicology studies, and ensuring comprehensive documentation of all preclinical findings to support an IND application.
How do you assess the stability and delivery efficiency of mRNA in preclinical studies?
Stability is assessed through degradation studies under various conditions, while delivery efficiency is evaluated using cell-based assays and animal models to measure how effectively the mRNA reaches target cells and produces the desired protein.
What are the common toxicological studies performed for mRNA therapies?
Common toxicological studies include acute and chronic toxicity assessments, genotoxicity tests, reproductive toxicity studies, and immunotoxicity evaluations to identify potential adverse effects and establish safe dosing ranges.
How is the immunogenicity of mRNA evaluated during preclinical studies?
Immunogenicity is evaluated by assessing the immune response to the mRNA and the encoded protein in animal models, measuring cytokine levels, antibody formation, and potential allergic reactions.
What are the key components of an mRNA therapeutic that need to be tested preclinically?
Key components include the mRNA sequence, the lipid nanoparticle (or other delivery system), and any additional stabilizing or targeting elements. Each component’s efficacy, stability, and safety must be evaluated.
How is the expression and functionality of the encoded protein measured in mRNA preclinical testing?
Expression and functionality are measured using techniques such as Western blotting, ELISA, and activity assays to quantify protein levels and assess its biological activity in cells and tissues.
What are the considerations for selecting delivery systems for mRNA therapeutics in preclinical research?
Considerations include the efficiency of mRNA encapsulation, protection from degradation, targeted delivery to specific cells or tissues, minimizing toxicity, and ensuring scalability for clinical use.
How do you ensure the scalability of preclinical testing results for mRNA therapies to clinical trials?
Scalability is ensured by developing robust manufacturing processes, performing thorough characterization studies, and validating analytical methods. Consistent and reproducible results in preclinical studies support the transition to clinical trials.
What methods are used to optimize the design of mRNA sequences for therapeutic use?
Methods include codon optimization to enhance translation efficiency, incorporating modified nucleotides to improve stability and reduce immunogenicity, and designing untranslated regions (UTRs) to regulate protein expression.
How do you evaluate the potential for off-target effects in mRNA preclinical testing?
Off-target effects are evaluated using bioinformatics tools to predict unintended interactions, in vitro assays to test for non-specific protein expression, and in vivo studies to monitor adverse effects in non-target tissues.
What advances in mRNA technology have improved the preclinical testing process?
Advances include improved lipid nanoparticle delivery systems, enhanced mRNA stability through chemical modifications, more accurate and predictive in vitro models, and sophisticated analytical techniques to measure mRNA and protein dynamics.
Let’s Talk About Your mRNA Program
Have questions? Need specific assays? Talk to an expert today about your mRNA needs.