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Nucleic Acid Drug Design Platform

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Based on a rigorous scientific approach and an open technology platform for nucleic acid drug design, as well as advanced instrumentation, CD Formulation can fulfill the industry's requirements for cutting-edge innovative nucleic acid drug development. It also offers nucleic acid drug development services for pharmaceutical companies and scientific research institutions.

Advantages of Nucleic Acid Drug Design Platform

Rapid and intuitive design of nucleic acid drugs is based on base sequences, utilizing simple preparation raw materials and processes, affordable production costs, allowing for a dramatic reduction in the cycle of drug development. This makes drug customization or personalized treatment protocols possible and enables the resolution of thorny issues plaguing the current pharmaceutical industry, such as rare diseases. The nucleic acid drug design platform was created to provide a full-process solution from sequence design to drug optimization using technologies such as computational biology, big data analysis, and machine learning.

Fig.1 Nucleic acid drug design offers various benefits Fig.1 Advantages of nucleic acid drug design. (CD Formulation)

Our Nucleic Acid Drug Design Platform

Our nucleic acid drug design platform is developed in-house by CD Formulation for the precise design of small nucleic acids and mRNA drugs. Utilizing our precision design platform and various high-throughput technologies, lead molecules can be efficiently validated in vitro or in vivo within a short period for a specific disease or target.

Target Identification and Validation Platform for Nucleic Acid Drugs

Target Screening Platform for Nucleic Acid Drugs

Sequence Design Platform for Nucleic Acid Drugs

Our Strategies for Designing Nucleic Acid Drugs

With state-of-the-art instrumentation and rich experience in nucleic acid drug development, CD Formulation can provide many types of service solutions according to clients' existing R&D progress.

Structure Optimization

In order to optimize the secondary and tertiary structures of mRNAs and enhance their stability and translation efficiency, we have adopted a series of computational biology methods. Specifically, we introduced stabilizing elements such as cap structures and poly(A) tails, aiming to significantly enhance their resistance to nuclease degradation. In addition, the biological stability of mRNAs can be enhanced by applying chemical modification techniques such as 2'-O-methylation and locked nucleic acids.

Sequence Optimization

In the process of optimizing the coding sequence, we have fine-tuned the frequency of codon usage to align with the preference of host cells, thereby enhancing the protein expression level. For the crucial functional regions of target genes, we design sequences with a high degree of specificity to enhance the precision and effectiveness of therapeutic effects.

Immune Escape

With the cutting-edge technology of bioinformatics, we can accurately predict and avoid key sequence regions that may trigger immune responses to ensure the safety and effectiveness of research and applications.

Highlights of Nucleic Acid Drug Design Platform

We can deliver candidate molecules, sequences, and delivery vehicles validated through in vitro and in vivo experiments based on specific diseases or targets provided by our customers. Our services leverage multiple high-throughput screening and validation technology platforms.
Combined with artificial intelligence technologies, the platform can extract valuable information from the large amount of data obtained from experiments to guide design optimization and plan further experiments.
The platform provides comprehensive safety assessment functions, including immunogenicity, toxicity, and pharmacokinetic assessments, enabling the early identification of potential safety issues.

Custom Nucleic Acid Drug Development Services

CD Formulation's nucleic acid drug sequence design platform enables the development of custom nucleic acid drugs by accurately identifying and optimizing nucleic acid sequences to meet specific therapeutic needs through algorithms and bioinformatics tools.

Custom Small Nucleic Acid Synthesis

The process of developing small nucleic acid drugs on our platform includes bioinformatics screening of candidate sequences, optimization of algorithms to improve specificity and stability, in vitro and in vivo validation of efficacy and safety, and structural modification and pharmacokinetic optimization to enhance the overall potential for clinical application.

Custom mRNA Synthesis

Our platform utilizes bioinformatics to analyze mRNA sequence properties, optimize the stability and expression efficiency of coding sequences, and design highly efficient mRNA drug candidates.

Nucleic Acid Modification Services

When modifying nucleic acid drugs, our platform utilizes chemical modification techniques to alter specific positions of the nucleic acid chain. This process aims to enhance their stability, specificity, and half-life in vivo, while reducing immunogenicity and toxicity.

Publication Data

Technology: Developing RNA drugs through machine learning (ML) technology

Journal: Artificial intelligence chemistry

IF: 14.05

Published: 2024

Results:

RNA molecules play multifaceted functional and regulatory roles within cells and have attracted much attention as promising therapeutic targets in recent years. With the impressive achievements of Artificial Intelligence (AI) in fields as diverse as computer vision and natural language processing, there is an increasing need to utilize the potential of AI in computer-aided drug design (CADD) for the discovery of novel drug compounds targeting RNA. Although machine learning (ML) methods have been widely used for the discovery of small molecule compounds targeting proteins, the application of ML methods to the modeling of interactions between RNA and small molecule compounds is still in its infancy. Compared with protein-targeted drug discovery, the main challenge for ML-based RNA-targeted drug discovery comes from the scarcity of available data resources. With the growing interest in RNA-small molecule interactions and the development of research databases focusing on RNA-small molecule interactions, the field is expected to grow rapidly and open up a new avenue for disease treatment. In this review, the authors aim to provide an overview of recent advances in the computational modeling of RNA-small molecule interactions in the context of RNA-targeted drug discovery, with a special emphasis on approaches employing ML techniques.

Fig. 2 Workflow of RNA-targeted drug discovery. Fig. 2 Structure-based RNA-targeted drug discovery workflow. (Zhou Y, et al., 2024)

CD Formulation is your specialized partner for nucleic acid drug development. We offer a comprehensive range of services from drug development to scale-up to expedite your project. Our goal is to assist our clients in saving time and money while delivering top-notch results. Contact us and we will customize a solution for your project.

References

Zhou Y, Chen S J. Advances in machine-learning approaches to RNA-targeted drug design. Artificial intelligence chemistry, 2024: 100053.

How It Works

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

STEP 2

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STEP 3

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

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