High Throghuput Screening Solution For small nucleic acid drugs -Foregene

Introduction

On March 7, 2023, AstraZeneca Pharmaceuticals and Ionis' ASO drug "Eplontersen" applied for marketing in China for the treatment of hereditary transthyretin amyloidosis. The drug has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) in early 2022.

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Types and development status of small nucleic acid drugs

Types and Development Status of Small Nucleic Acid Drugs

?Since the discovery of mRNA in 1961, to ASO, aptamers, RNAi, and delivery systems, small nucleic acid drugs have gradually moved from imagination to reality, becoming the third largest class of drugs after small molecule drugs and antibody drugs, leading the new A wave of pharmaceuticals.

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Small nucleic acid drugs, that is, oligonucleotide drugs, are short-chain nucleic acids composed of a dozen to dozens of nucleotides in series, acting on pre-mRNA or mRNA, and achieving the purpose of disease treatment by interfering with the expression of target genes. It mainly includes RNAi drugs (siRNA, miRNA) and antisense nucleotide (ASO) drugs, as well as nucleic acid aptamers and other types of short RNA fragments (saRNA, sgRNA, tRNA fragments, etc.).

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Advantage

Based on its characteristic of targeting pre-mRNA or mRNA, small nucleotide drugs can realize the treatment of diseases at the RNA level, and there are more optional drug targets, which have the following multiple advantages:

?At first, the bottleneck in the development of small nucleic acid drugs was how to avoid the degradation of nucleases in the receptor body, and successfully enter the cell to regulate the expression level of RNA or the process of protein translation. With the breakthrough of chemical modification technology and the emergence of GalNac delivery system, the instability of small nucleic acid drugs and the lack of effective delivery system have been initially solved. Small nucleic acid drugs are based on the active, digital design and synthesis mode of disease target gene sequences, which greatly shortens the unavoidable screening cycle of traditional drugs. The scale of the global related market has grown rapidly since 2016, and has grown from more than US$10 million at the beginning to US$3.784 billion in 2022, with an average annual growth rate of more than 12% in the past three years, becoming the forefront of the global drug research and development field.

Global Small Nucleic Acid Drug Market Size Statistics (2016-2022, unit: USD 100 million)

Key issues and challenges in small nucleic acid drug development

Key issues and challenges in the development of small nucleic acid drugs

?Different problems in the development of small nucleic acid drugs need to be solved by corresponding technologies. According to the small nucleic acid drug development process, the core technology is divided into RNA design, chemical modification, delivery, synthesis and preparation technology. The most important core technology is to efficiently and accurately obtain effective and specific molecular sequences; the most critical core technology is small nucleic acid drug delivery technology, which enhances molecular stability and targeting capabilities, reduces dosage and side effects.

?With a large amount of capital entering the small nucleic acid drug R&D industry, given the diversity and complexity of target gene sequences, how to quickly and accurately screen nucleic acid fragments with medicinal value has become the primary task of R&D. Small nucleic acid drugs target specific RNA fragments in cells, so the significant impact on the up-regulation or down-regulation of their expression levels is an important indicator for drug effectiveness screening.

?For the traditional research method of up-regulation or down-regulation of cell gene expression level, it is necessary to continue to culture cells to a certain order of magnitude after drug treatment, collect cells and purify RNA, and then obtain CT values by fluorescent quantitative PCR for analysis and comparison. This process is limited by RNA purification methods and throughput limitations, and consumes a lot of time, manpower, and funds in the process of cell culture and nucleic acid extraction, and cannot quickly and efficiently obtain data for analysis. At the same time, due to relatively many experimental links, the data uniformity and stability cannot meet the requirements of precise screening.

?Foregene

Based on the direct PCR/qPCR technology platform, Foregene Biotech has brought an innovative and efficient " 5H " screening solution - Cell Direct RT-qPCR kit, which can process cells in situ on the culture plate to obtain RNA templates for reverse transcription, saving a lot of operations and time, and can be adapted to automation, efficiently and accurately obtain the results of cell GOI expression levels, and quickly determine nucleic acid fragments with medicinal value!

?H1

H1-High Efficiency: The whole plate RNA template preparation can be completed in 7 minutes (5mins cleavage + 2mins termination), no need for large instruments. The FAST amplification program of various models can be used, and the entire screening process can be completed within 90 minutes at the fastest, saving 80% of the time compared with traditional methods!

?5min in situ cleavage of the plate wells to release RNA, 2min to terminate the reaction

The resulting lysate can be used as a template for subsequent RT-qPCR reactions

?H2

H2-High Precision High Precision: Fully automatic operation can be realized, the uniformity of the whole board is good, and the precision is orders of magnitude higher than the traditional method.

H3

H3 - High Throughput: A maximum of 384 wells of gene expression screening can be performed at one time, which is more than 4 times higher than traditional methods.

H4

H4-High adaptability: It is suitable for the expansion and screening of suspension cells and adherent cells, and is suitable for more than 10 commonly used cell types. The kit has a very low detection limit, and the 10 - 10^6 cell level can achieve accurate amplification.

?H5

H5-High Accuracy High Accuracy: UDG anti-pollution system can be used; at the same time, RT-qPCR reaction enzyme inhibitors are extremely resistant, and can ignore the residual interference of the medium, making the screening results more reliable.

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Contact: [email protected]

WhatsApp: 0086-15281067355

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Time-limited free samples trial application registration period: June 20, 2023 to July 7, 2023.

Free trials: E-mail [email protected], send an e-mail to register directly, and you can try it out after passing the review (limited to 15 copies, and each research group is limited to 1 person to apply for 1 copy)

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