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Target Identification and Validation Platform for Nucleic Acid Drugs

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CD Formulation's team has extensive experience in gene editing, employing various approaches from single gene to whole genome editing. The team also possesses strong capabilities in bioinformatics, pathway analysis, and biofunctional analysis. Additionally, our research team is confident in offering customized one-stop target identification and validation services to help you achieve your goals in a high-quality, reliable, and cost-effective manner.

Advantages of Target Identification and Validation for Nucleic Acid Drugs

The first stage of nucleic acid drug development is target discovery, also known as target identification and validation. This stage involves identifying potential targets for therapeutic drugs to treat a specific disease or condition that currently lacks treatment or has insufficient therapeutic options. The target may be known, hypothesized, or entirely new. In target-based screening, each candidate target undergoes validation through various experimental methods, including modulating the target to establish its direct or indirect association with the target disease or condition.

Fig.1 Advantages of target identification and validation platforms for nucleic acid drugs. (CD Formulation)

Construction of Target Identification and Validation Platform for Nucleic Acid Drugs

There are various methods for constructing target identification and validation platforms for nucleic acids, depending on the needs and goals of the study. Some common methods include:

Technologies	Items
Nucleic Acid Probe Technology	FISH (Fluorescence In Situ Hybridization) is a technique used for detecting target DNA or RNA through hybridization with fluorescently labeled DNA or RNA probes. Labeled Nucleic Acid Probes - Probes are conjugated to fluorescent dyes, radioisotopes, enzymes, or other markers, typically through enzymatic digestion or chemical synthesis.
Nucleic Acid Amplification Technology	PCR (Polymerase Chain Reaction) involves amplifying target DNA or RNA fragments using specific primers. qPCR (quantitative PCR) and RT-qPCR (reverse transcription quantitative PCR) are used to accurately quantify target nucleic acids.
CRISPR-Cas Technology	CRISPR-Cas9 is used for gene editing and identifying targeted DNA. CRISPR-dCas9 is a modified Cas9 protein that does not cut DNA but is used to target and label specific gene loci.
High-throughput Sequencing Technology	Various techniques such as RNA-seq, ChIP-seq, Methyl-seq, etc., are utilized to comprehensively identify and validate target nucleic acid sequences in genomes, transcriptomes, and epigenomes.
Mass Spectrometry	Mass Spectrometry Sequencing utilizes mass spectrometry to directly determine nucleic acid sequences.

Highlights of Nucleic Acid Drug Target Identification and Validation Platform

Modern nucleic acid detection platforms enable the rapid detection of target sequences in a large number of samples with high sensitivity, capable of detecting very small amounts of target sequences.
Many nucleic acid assays, such as CRISPR/Cas and aptamer technology, can identify target sequences with high specificity, thereby reducing false alarm rates.
The platform can be adapted as needed for a variety of application scenarios, covering all areas from basic research to clinical diagnostics.
Nucleic acid detection platforms have been automated and standardized to greatly reduce human error, and enhance reproducibility and accuracy.

Custom Nucleic Acid Drug Development Services

CD Formulation can identify and characterize promising therapeutic targets using a variety of methods. We are constantly developing new tools and techniques to validate targets more effectively and pinpoint promising leads at the onset of drug development programs.

Custom Small Nucleic Acid Synthesis

Using a target identification and validation platform for nucleic acid drugs, we can rapidly discover key regulatory targets associated with diseases. We can design small nucleic acid sequences with optimized affinity and targeting properties for these targets. Simultaneously, we will develop efficient nano-delivery systems and establish standardized production processes.

Custom mRNA Synthesis

We utilize the target identification and validation platform of nucleic acid drugs to develop mRNA drugs. Initially, we identify the gene expression variances of specific diseases through high-throughput screening technology. Subsequently, potential therapeutic targets are screened using informatics tools and experimental data. Finally, advanced synthesis and delivery technologies are employed to transport the designed mRNAs to the target cells for precision therapy.

Publication Data

Technology: Drug-target identification and prioritization through the Open Target Platform

Journal: Nucleic acids research

IF: 16.971

Published: 2021

Results:

The Open Target Platform provides users with a searchable knowledge base and user interface to help systematically identify targets and prioritize drug discovery based on underlying evidence. The platform is publicly available and its underlying code is open source. The authors integrated new evidence from key datasets, including prioritized targets identified by genome-wide CRISPR knockout screens in 300 cancer models (item scoring), and evidence from GWAS/UK BioBank statistical genetic analyses from the Open Targets Genetics Portal. The authors improved the evidence scoring framework to improve target identification. To help prioritize targets and inform the potential impact of modulating specific targets, the authors added an assessment of post-marketing adverse drug reactions and new curated information on target tractability and safety. The authors also developed the user interface and backend technologies to improve performance and usability. In this paper, the authors describe the latest enhancements to the platform to address the fundamental challenge that developing effective and safe drugs is both difficult and expensive.

Fig. 2 Drug-target identification and prioritization through the open target platform. Fig. 2 Overview of the open targets platform. (Ochoa D, et al., 2021)

CD Formulation specializes in identifying and validating targets for different therapeutic indications. We develop tailored models with specific readouts and utilize bioinformatics, bulk and single-cell next-generation sequencing, CRISPR/Cas9, and other cutting-edge technologies. Contact us and we can support your program with assayability and druggability assessments for your nucleic acid drug development.

References

Ochoa D, Hercules A, Carmona M, et al. Open Targets Platform: supporting systematic drug-target identification and prioritisation. NAR. 2021, 49(D1): D1302-D1310.

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