Nucleic Acid Drug Toxicology Analysis

As a company specializing in advanced therapeutic drug discovery, CD Formulation understandS the critical role of toxicology analysis in nucleic acid drug development. With an experienced toxicology research team, advanced analytical instruments, and technology platforms, we are able to provide our clients with comprehensive toxicology solutions for nucleic acid drugs.

About Toxicology Analysis of Nucleic Acid Drugs

Toxicology is the study of the damage that drugs may cause to the organism under certain conditions. It is an essential aspect of nucleic acid drug development, crucial for the initial clinical trials of new drugs, and holds significant importance for identifying and managing clinical risks. The toxicity assessment of nucleic acid drugs typically involves safety pharmacology experiments, general toxicology experiments, genotoxicity experiments, reproductive toxicity experiments, and carcinogenicity experiments. Technical guidelines encompass relevant guidelines on drug safety evaluation from the U.S. FDA and ICH. A systematic toxicological analysis of nucleic acid drugs aids in recognizing and managing potential safety risks, serving as a fundamental basis for optimal drug design, preclinical evaluation, and clinical application. This process ensures that these innovative therapeutic approaches are safe and effective, ultimately benefiting patients.

Fig.1 In vitro models of target-organ toxicity. (Pognan F, et al., 2023)

Explore Our Nucleic Acid Drugs Toxicology Analysis Services

Based on FDA expectations and regulatory guidance, our drug toxicology testing will meet all of your specific requirements. We have years of experience providing toxicology, pharmacology, and analytical services to the nucleic acid formulation industry. With a team of experts who have extensive experience in nucleic acid formulation testing, we can assist you with everything from evaluating drug development to scale-up. Our services include:

Safety Pharmacology Studies

The program includes safety pharmacology experiments on the respiratory, central, and cardiovascular systems in a variety of animal species, such as mice, rats, and monkeys. We can conduct in vitro hERG experiments to further evaluate the cardiovascular risks that the drug may induce. Apart from the three core systems mentioned above, additional hepatic and renal safety pharmacology experiments also need to be considered because some nucleic acid drugs are metabolized by the liver and excreted by the kidneys, respectively.

General Toxicology Studies

General toxicology encompasses single and repeated dose studies of various durations, including up to 2-year carcinogenicity studies. Depending on client and regulatory requirements, we are capable of conducting both GLP and non-GLP studies of different species, scale, frequency, intricacy, and adherence to regulations.

In addition to traditional assays, we can analyze the efficacy endpoints of nucleic acid drugs to further confirm the drug's ability to bind to the target mRNA in tissues and generate the appropriate pharmacological effect during extended administration.

Genotoxicity Studies

Genotoxicity studies are crucial components of preclinical safety evaluations of drugs and are closely linked to other studies, particularly toxicology assessments like carcinogenicity tests and reproductive toxicity tests. They serve as a vital step for drugs to progress into clinical trials and marketing. Our genotoxicity study services encompass the Ames test, Mini-Ames test, chromosomal aberration test, and micronucleus test.

Reproductive Toxicity Studies

Nucleic acid drugs typically follow a segmented reproduction testing strategy, which includes fertility and early embryo developmental toxicity tests, embryo-fetus developmental toxicity tests, and perinatal developmental toxicity tests. The animal species chosen are predominantly mice, rats, and rabbits, and the dosing method generally mirrors the intended clinical route of administration. Furthermore, toxicology experiments on young animals are also conducted based on the target population.

Carcinogenicity Studies

In accordance with ICH S1A guidelines, we provide carcinogenicity studies for nucleic acid drugs. Long-term carcinogenicity studies are usually carried out in rats over a 2-year period, while 6-month carcinogenicity studies are conducted in transgenic mice using the clinically recommended route of administration.

Our Technology Platforms for Nucleic Acid Drugs Toxicology Analysis

Our Workflow for Nucleic Acid Drugs Toxicology Analysis

Fig.2 Flow chart of nucleic acid drugs toxicology analysis. (CD Formulation)

• Sample Preparation - We will collect the nucleic acid drug samples provided by our clients and prepare them at various concentration gradients for subsequent experimental analysis.
• In Vitro Cytotoxicity Assessment - This phase involves cell culture and the execution of assays such as MTT, CCK-8, and LDH to evaluate the cytotoxic effects of nucleic acids on cells.
• Genotoxicity Assessment - To evaluate the genotoxicity of nucleic acid drugs, we will conduct the Ames test, the mouse micronucleus test, and DNA damage assays.
• Reproductive Toxicity Analysis - The effects on reproductive health are assessed by quantifying reproductive organ weights, sperm parameters, and sex hormone levels.
• Carcinogenicity Assessment - Evaluating the carcinogenic potential requires long-term animal studies and cell transformation assays to ascertain the risk of carcinogenicity associated with nucleic acid drugs.
• Data Analysis - We will conduct a statistical analysis of all collected data and compile a comprehensive toxicology report to provide our clients with a thorough evaluation of the results.

Why Choose Us for Nucleic Acid Drugs Toxicology Analysis?

• Meet Regulatory Requirements in Multiple Countries
  Our analytical methods and data meet the standards and guidelines of major regulatory agencies such as the US FDA and the European EMA.
• Comprehensive Analytical Coverage
  We can conduct a comprehensive and systematic evaluation and analysis of all toxicity endpoints of nucleic acid drugs, including genotoxicity, reproductive toxicity, carcinogenicity, and more.
• Data Analysis Optimization
  We have advanced statistical analysis tools that enable us to delve deeper into the experimental data and analyze it to provide customers with more accurate and comprehensive toxicology analysis reports.
• Quality Assurance
  We strictly comply with GLP/GCP and other quality management systems to ensure the reliability and reproducibility of the entire analytical process and data.

With our professional team and advanced technology, CD Formulation is committed to providing high-quality and reliable toxicological analysis of nucleic acid drugs for our clients. This will contribute to the safe and effective application of this emerging therapy for the benefit of patients. Contact us and we can help you with drug development.

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How It Works

STEP 1

Submit a service request describing your specific research or development need.

STEP 2

We'll email you to provide your quote and confirm order details if applicable.

STEP 3

Execute the project with real-time communication, and deliver the final report promptly.