Biomedical Technology Great Leap Forward: The RNA-Base Era Comes Quietly

2018/09/28 14:38
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It is estimated that the market for new biopharmaceutical technology will reach 1.2 billion in 5 years: RNA-Base is more promising than DNA-Base
RNA-based biopharmaceuticals are a relatively new class of technologies, including the development of new therapies and vaccines based on RNA, which are targeted at the treatment and prevention of chronic and rare diseases such as cancer. Diabetes, tuberculosis, and certain cardiovascular diseases. According to market research, DNA and RNA therapy are expected to grow at a compound annual growth rate of 12%, and the market size is expected to reach $1.2 billion by 2020.
There are currently 700 drug development projects using DNA and RNA therapy, but most (about 430) projects are still in the early stages (preclinical), and 35% of these projects target tumors. Worldwide, approximately 160 companies and 65 universities are developing RNA-Base therapies. There are currently 12 mRNA vaccines under development, seven of which are developed by CureVac. According to current market trends, the RNA therapy market seems to be more promising than DNA therapy.
In tumor-targeted projects, it is dominated by RNA interference (RNAi) and antisense RNA technology. RNAi is a gene silencing technology that inhibits gene expression by targeting and destroying specific messenger RNA molecules (mRNA). Antisense RNA refers to an RNA molecule complementary to mRNA, and also includes an RNA molecule complementary to other RNA. Since ribosomes are unable to translate double-stranded RNA, antisense RNA binds specifically to mRNA and inhibits translation of the mRNA.
A major challenge in the commercialization of RNA-Base therapies is toxicity and delivery of the drug. The ideal drug delivery mainly includes the following characteristics: biocompatibility, avoiding the influence of nuclease, controlling distribution (permanence and location of drug), fullness of activity and safety.
RNA-Base therapy
RNAi works by silencing or shutting down genes. RNAi was first discovered in plants in the 1990s and is called "gene silencing." In 2001, studies showed that mammalian cells have similar mechanisms. Two RNAs are involved in RNAi technology: small interfering RNA (siRNA) and micro RNA (miRNA).
siRNA, also known as short interfering RNA or silencing RNA, triggers the degradation of messenger RNA, which requires only a small amount of siRNA to effectively silence gene expression. siRNA is formed by Dicer cleavage of double-stranded RNA, which is usually injected into cells by microinjection or transfection. Many companies currently offer siRNA delivery reagents to simplify the process. A miRNA is a specific non-coding RNA with a total length of 19-25 nucleotides. The miRNA inhibits translation of the target gene by acting as an RNA silencing complex guide chain of the target mRNA. When siRNA silencing requires precise matching of targets, miRNAs can function through imperfect base pairing.
RNA-Base vaccine
An immune response can be induced by direct injection of a vaccine that encodes an RNA molecule that targets the antigen and is absorbed by the antigen-presenting cells. Typically, mRNA vaccines are produced by enzymatic engineering and can be tightly controlled for immunogenicity, pharmacokinetics, and dosage, and mRNA can be used to optimally optimize codon usage and optimize antigenic properties and vaccine stability. Clinical trials have shown that direct delivery of mRNA (in liposomes) produces a distinct protective immune response, and mRNA can also act as an adjunct to stimulate innate immune responses. The RNA-Base vaccine can be developed, manufactured and managed in a matter of weeks and is a potential weapon to lift the threat of future epidemics.
RNA-Base Biopharmaceutical
The production of RNA-Base biopharmaceuticals requires special care because chemical instability and endonuclease are ubiquitous (especially in the laboratory) making these drugs very sensitive. The principles of RNA-Base biopharmaceuticals are as follows:
Biomedical Technology Great Leap Forward: The RNA-Base Era Comes Quietly
The siRNA was first synthesized by chemical synthesis, but it can also be synthesized in the laboratory by in vitro transcription. Purification of mRNA typically involves concentration, precipitation, extraction, and separation. The 5kD membrane is typically used for mRNA concentration and permeation processes, and because siRNA is smaller than mRNA, a 1 kD membrane is used to trap siRNA products. Traditional ion exchange media (IEX), especially anion exchange (AEX), remains the most popular technique for delivering purified RNA and RNA hybrids.
The biggest obstacle to RNA-Base biopharmaceuticals is how to deliver therapeutic drugs to diseased cells. More and more research is currently focused on combining RNA therapy with other systems to increase the efficiency of RNA-Base drug delivery and absorption. The most popular strategy today is the use of lipid delivery systems.
in conclusion:
RNA-Base biopharmaceuticals are a relatively new technology and are very promising and increasingly used for the treatment and prevention of chronic and rare diseases. Use this method to develop new therapies and vaccines with extra care because RNA is easily degraded. The biggest challenge in the commercialization of RNA-Base therapy is the two major problems of toxicity and how to effectively deliver drugs. However, new technologies are emerging to overcome these difficulties.