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Sullivan released "Research Report on Current Situation and Future Development of Nucleic Acid Drug Market Industry"

 Last Update: 2022-08-15
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Nucleic acid drugs are one of the most promising frontiers in the future of biopharmaceutica.

Frost & Sullivan ("Sullivan") officially released the "Research Report on the Current Situation and Future Development of the Nucleic Acid Drug Market Industry" (hereinafter referred to as the "Report")

In the historical process of fighting diseases, traditional medicines have used "proteins" as drug targe.

mRNA drugs: Shining bright in the new crown epidemic, the future application space is broad

0 mRNA drug application scenarios

According to the use and type of drugs, mRNA drugs have three main application directions, including infectious disease vaccines (prophylactic vaccines), tumor treatment (therapeutic vaccines), and protein replacement therapy (therapeutic drug.

There are three main application directions of mRNA drugs, including infectious disease vaccines (prophylactic vaccines), tumor treatment (therapeutic vaccines), and protein replacement therapy (therapeutic drug.

mRNA technology has obvious advantages and will become an important choice for the development of new vaccin.

0 mRNA drug development challenges and key technologies

Due to the large molecular weight, strong immunogenicity and easy degradation of mRNA, there are many obstacles in the development of mRNA dru.

0 mRNA drug delivery system

The main delivery carrier technologies for mRNA drugs include liposome nanoparticles, protamine carriers, and polymer carrie.

Currently, liposome nanoparticles are the most widely used delivery syste.

The continuous improvement of the security and stability of LNP is the key to future resear.

0 mRNA vaccine global market size

mRNA vaccines are general-purpose platform-based technologies that provide a flexible, fast, large-scale, and cost-effective production process, with obvious advantages in the face of the threat of a new crown pandem.

According to the phase III clinical results, the mRNA new crown vaccine showed superior protective efficacy and activation ability to the immune system than other new crown vaccin.

The global sales revenue of the two mRNA new crown vaccines is as high as 57 billion US dollars, both far exceeding the sales of other new crown vaccin.

In the short term, the main contributor to the future market of mRNA products will still be the new crown vacci.

Small nucleic acid drugs: rapid development in recent years, large room for expansion of indications

0 Classification and treatment principle of small nucleic acid drugs

Small nucleic acid drugs, namely oligonucleotide drugs, are short-chain nucleic acids consisting of dozens 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 gen.

0 Small nucleic acid drugs that have been marketed around the world

At present, a total of 14 small nucleic acid drugs have been approved for marketing in the world (3 early drugs have been delisted), including 9 ASO drugs, 4 siRNA drugs and 1 nucleic acid aptam.

After years of development of small nucleic acid drugs, blockbuster products have emerg.

0 Advantages of small nucleic acid drugs

The emergence of small nucleic acid drugs makes it possible to actively design drug sequences to target and silence disease genes, which has multiple advantages compared with traditional dru.

Abundant candidate targets and wide distribution of indications

Theoretically, any disease caused by the overexpression of a specific gene can be treated by small nucleic acid drugs, which provides a wealth of candidate targets for the development of small nucleic acid drugs, including many targets that cannot be made by traditional dru.

Simple drug design and short development cycle

Small nucleic acid drugs are the field of greatest benefit from the development of genomics and functional genomics so f.

The design of small nucleic acid drugs can realize digital design directly targeting disease gene (mRNA) sequences, thus getting rid of the large-scale screening cycle of traditional drugs, enabling drug design to be actively carried out, and the success rate of research and development is relatively hi.

Strong targeting specificity

Small nucleic acid drugs realize gene expression regulation from the post-transcriptional level, can specifically act on the mRNA transcribed by pathogenic genes, regulate the expression of pathogenic genes from the upstream of the disease, and can achieve sequence specificity at the single-base level, It has the characteristics of "targeted" and "treatment of symptoms and root cause.

In addition, the continuous innovation of nucleic acid drug delivery systems can effectively deliver nucleic acid drugs to target organs and truly achieve targeted drug delive.

long-acting drug

The target of small nucleic acid drugs is mR.

Taking siRNA drugs as an example, when the target mRNA is degraded, the RNA-induced silencing complex can work cyclically and participate in the next round of target mRNA degradati.

Therefore, small nucleic acid drugs can achieve long-term effects in cel.

In addition, the chemical modification technology of nucleic acid drugs is constantly innovating, including phosphate backbone modification, sugar modification and base modification, e.

, which can improve the stability and half-life of nucleic acid drugs, make the drug effect longer, and greatly improve the compliance of patients with treatme.

0 Challenges and key technologies of small nucleic acid drug development

Small nucleic acid drugs face the problem of how to efficiently enter target cells, release them into the cytoplasm, and play an efficient and long-term effe.

Different problems in the development of small nucleic acid drugs need corresponding technologies to be solv.

According to the sequence of the small nucleic acid drug development process, the core technologies are divided into design, chemical modification, delivery, synthesis and formulation technologi.

The most critical core technology is nucleic acid drug delivery technolo.

Drug delivery plays an important role in protecting RNA structure, enhancing targeting ability, reducing administration dose, and reducing toxic and side effec.

Small nucleic acid drugs face the problem of how to efficiently enter target cells, release them into the cytoplasm, and play an efficient and long-term effe.

The most critical core technology is nucleic acid drug delivery technolo.

Drug delivery plays an important role in protecting RNA structure, enhancing targeting ability, reducing administration dose, and reducing toxic and side effec.

0 Small nucleic acid drug delivery system

Small nucleic acid delivery systems can be divided into naked RNA-modified delivery systems, liposome nano-delivery systems, conjugated-linked delivery systems (small molecule ligands, antibodies and other molecules) and other delivery systems (polymer matrices, exosomes, viruses) transfection, e.

Among the 14 small nucleic acid drugs that have been marketed, 10 small nucleic acid drugs do not use delivery systems, 1 is nucleic acid aptamer, and the remaining 9 are ASO dru.

This is mainly due to the small molecular weight of ASO drugs and their ability to enter cel.

Strong, especially ASO drugs can enter the central nervous system without the need for a delivery system after intrathecal injection, so there are many ASO drugs for central nervous system diseases currently being develop.

The remaining 4 siRNA drugs all used the delivery system, 1 used the lipid nanoparticle delivery system, and 3 used the GalNAc conjugated ligation delivery syst.

The realization of the GalNAc conjugated delivery system is a major breakthrough in the development of small nucleic acid dru.

This technology solves three major pain points in the development of small nucleic acid drugs: poor targeting, serious off-target effects, and poor stabili.

major breakthrou.

GalNAc is linked to siRNA and administered by subcutaneous injecti.

It binds to the ASGP receptor on the surface of liver cells and targets the liv.

The delivery efficiency is greatly improved, and the effect lasts for several mont.

Due to its excellent targeting properties for the liver, a number of small nucleic acid drugs for liver diseases are being develop.

In addition, GalNAc also has affinity for TTR and other targets in the cardiovascular system, and it also has broad application prospects in the field of cardiovascular system diseas.

0 Global market size of small nucleic acid drugs

The first three small nucleic acid drugs have been delist.

With the launch of two ASO drugs in 2016, the silence of the drug market for many years has been brok.

The global market size of small nucleic acid drugs has grown from US$10 million in 2016 to US$25 billion in 2021, with a compound annual growth rate of 21
In the future, with the continuous listing of small nucleic acid drugs in the clinical stage, especially drugs with indications for larger patient groups, such as potential curative drugs for hepatitis B, will further drive the rapid development of the mark.

The global market size of small nucleic acid drugs has grown from US$10 million in 2016 to US$25 billion in 2021, with a compound annual growth rate of 21
In the future, with the continuous listing of small nucleic acid drugs in the clinical stage, especially drugs with indications for larger patient groups, such as potential curative drugs for hepatitis B, will further drive the rapid development of the mark.

At present, the small nucleic acid drugs on the market are all ASO and siRNA dru.

ASO drugs are listed early, and their commercialization is more mature, and their share in the overall small nucleic acid drugs is relatively high, reaching nearly 8
In terms of indications, spinal muscular atrophy is currently the most successful indication for commercializati.

In 2021, there is only one drug for this indication, but it has contributed nearly 2 billion US dollars in sal.

Other indications include Duchenne muscular dystrophy, e.

Generally speaking, the current marketed products are mainly in the field of rare disease indicatio.

The successful commercialization also fulfills the original intention of solving clinical needs and pain points, but it also reveals from the side that the current The current situation that the small nucleic acid drug market lacks indications with a large patient populati.

At present, the products on the market are mainly in the field of rare disease indicatio.

The successful commercialization also fulfills the original intention of solving clinical needs and pain poin.

However, it also reveals from the side that the current small nucleic acid drug market lacks indications with large patient grou.

It is worth noting that due to the unique mechanism of action of small nucleic acid, it has greater development potential and clinical value in many indicatio.

There are many clinical trials targeting cancer, diabetes, hepatitis B and other major diseases in the clinical research pipeline, which will be extremely importa.

Dadi makes up for the lack of commercialized heavyweight varieties and supports the future development of the small nucleic acid drug mark.

The "Report" sorts out domestic and foreign companies that focus on the field of nucleic acid drugs, showing differentiated technological competitive advantag.

The following is the introduction of some nucleic acid drug companies:

Sirnaomics: Founded in 2007, headquartered in Gaithersburg, Maryland, USA, it currently has an R&D center in Suzhou, China, and a production base in Guangzhou, Chi.

The research leader is also the first biotechnology company in Hong Kong that focuses on the application of RNA technology for the development and creation of new dru.

The company has a globally exclusive peptide nanoparticle (PNP) delivery platform and uses it to develop innovative biopharmaceutical drugs for a variety of disease indications, including oncology, fibrotic diseases and disorders, viral infections and metabolic diseas.

The current core product, STP705, is the first drug candidate to achieve positive Phase IIa clinical results in oncolo.

Sirnaomics:

Aibo Bio: Founded in 2019, it is an innovative biopharmaceutical company focusing on messenger ribonucleic acid (mRNA) drug research and developme.

The company has industry-leading mRNA platform technology with independent intellectual property rights, including nano-liposphere delivery technology for efficient delivery of mRNA drugs, mRNA design, synthesis and modification technology to ensure the stability, safety and efficacy of mRNA molecules, as well as the realization of Dynamic precision mixing technology for industrial production of delivery syste.

At present, the company's pipeline covers the fields of infectious disease prevention and tumor immuni.

Among them, the mRNA vaccine developed for COVID-19 has entered the international multi-center phase III clinical study and obtained the drug production licen.

Aibo Bio:

Agna Bio: Founded in 2020, it is an RNA innovative drug company co-founded by RNAi discoverers, 2006 Nobel Prize winners and senior returnee scientis.

It has an experienced RNA drug research and development team and advanced RNA drug research and developme.

Platform and large-scale mRNA vaccine production line, with more than 20 invention patents in the field of R.

The company has independent intellectual property rights of GalPET liver-targeted RNAi and lipid nanoparticle (LNP) delivery core platform technolo.

In March 2022, the new crown mRNA vaccine developed by Agna Bio and Ribo Bio launched a Phase II clinical tri.

Agnar Bio:

Ribo Bio: Founded in 2004, it is a high-tech enterprise integrating R&D, production, sales and scientific research services with nucleic acid technology as the core and nucleic acid drug production services and nucleic acid scientific research products as the leadi.

The company provides nucleic acid drug product production and CMC pharmaceutical services for enterprises through oligonucleotide nucleic acid production, process development and analytical method resear.

The company currently has multiple platforms such as oligonucleotide drug production, analysis and testing, high-throughput sequencing, and new nucleic acid drug research and development, providing nearly 100 kinds of products and servic.

The company not only has the production capacity and large-scale production base of various chemically modified oligonucleotide drugs, but also provides the whole industry chain production of mRNA vaccines from plasmid manufacturing, raw materials and enzyme production, to mRNA production and LNP preparation and filli.

and development servic.

Ribo Bio:

Ribo Bio : Founded in 2007, it is an enterprise dedicated to the research and development of innovative small nucleic acid drugs, and a major pioneer of small nucleic acid technology and small nucleic acid pharmaceuticals in Chi.

Focusing on the characteristics of small nucleic acid drugs, the company has established a small nucleic acid innovation technology system integrating the entire technology chain and a small nucleic acid drug research and development platform covering the entire life cycle of small nucleic acid drugs from early discovery to industrializati.

The pipeline varieties based on the GalNAc delivery technology independently developed by the company have been advanced to the clinical stage, and the company has four products in the clinical research sta.

Based on unmet clinical needs, product pipelines under development are formed around multiple disease areas such as liver disease, cardiovascular disease, metabolism, ophthalmology and rare diseas.

The company will establish a European R&D center in 2022 and start an international layo.

Ribo Bio

In 2020, mRNA vaccines have gained widespread public attention, bringing about the rapid development of nucleic acid dru.

Nucleic acid drugs have gradually become the focus of biopharmaceutical investment and the research and development of pharmaceutical compani.

.

Xin Wang, Sullivan's global partner and president of Greater China, said, "The vaccination of hundreds of millions of doses of mRNA new crown vaccine has verified its efficacy, and it has also increased the overall nucleic acid drug research and development fev.

Nucleic acid drugs are suitable for rich and specific targe.

It has strong sexuality and can intervene in diseases from the sour.

At present, dozens of nucleic acid drugs have been launched in the world, and positive market feedback has been obtained, and the development of the nucleic acid drug industry is in the ascenda.

"

.

Xin Wang, Sullivan Global Partner and President of Greater China, said,

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