RNAi技術の世界市場-2023年～2030年

概要

世界のRNAi技術市場は2022年に16億米ドルに達し、2023-2030年の予測期間中にCAGR 10.3％で成長し、2030年には36億米ドルに達すると予測されています。

小さな核酸断片はRNA干渉（RNAi）という技術に採用され、転写後の遺伝子を沈黙させ、がんやその他の疾患を治療します。この技術を用いることで、結合に必要な酵素ポケットを持たないタンパク質をより標的特異的にすることができます。

この方法を用いることで、標的遺伝子を検証し、重要な疾患遺伝子を機能的に評価することができ、その結果、強力な医薬品を生み出すことができます。RNA干渉（RNAi）に基づく治療薬は、あらゆる分子クラスの疾患標的の特異的かつ強力な阻害剤を迅速に同定する強力な方法を提供します。

さらに、医薬品開発や農業バイオテクノロジーなど、さまざまな用途における正確な遺伝子サイレンシングに対する需要の高まり、これらの分野における研究の拡大が、RNAi技術の市場規模を押し上げています。

ダイナミクス

分子診断（がん）向け用途の拡大

遺伝的・代謝的問題の増加、分子診断用途の増加、がんの蔓延、創薬のための研究開発の拡大は、世界のRNAi技術市場の成長を促進する要因の一部です。がん治療におけるRNAiの利点には、進行期の腫瘍の効率的な増殖抑制、低コスト、優れた特異性などがあります。

薬剤耐性は、RNAiを使って異なる経路の複数の遺伝子を同時に阻害することで減少する可能性があります。例えば、研究者らは、SH3GL1をsiRNAで阻害することにより、EGFR/ERK/AP-1経路がP糖タンパク質の発現を低下させ、MDRを逆転させる可能性があることを発見しました。より効率的なデリバリー技術が開発されれば、RNAiは特定の患者に対する化学療法アジュバントとして、オーダーメイドの薬剤を提供するために使われるかもしれません。

さらに、酸化マンガン、酸化鉄、シリカ、有機金属骨格などの金属酸化物ナノ材料も、siRNAを用いた遺伝子治療に生産的に利用されています。siRNAキャリアとしての能力にもかかわらず、金属酸化物ナノ材料はその磁気特性により光線療法や画像化の可能性を持っており、磁気共鳴画像やセラノスティクス応用のための併用療法のための優れたバイオイメージングツールとなります。

医薬品承認の増加

siRNAは化学医薬品よりも特異性が高く、安全性が高いという利点があります。siRNAは効率の面でも適しており、siRNA医薬品の候補群を迅速かつ容易に開発することができます。

2022年12月現在、米国FDAによって5つのsiRNA医薬品が承認されています。Patisiran（Onpattro）、Givosiran（Givlaari）、Lumasiran（Oxlumo）、Inclisiran（Leqvio）、Vutrisiran（Amvutta）です。承認された5つのsiRNA薬剤はすべて肝臓で発現するmRNAを標的としています。動物に投与されたsiRNAは、外来物質を解毒する重要な臓器である肝臓に速やかに蓄積されるからです。しかし、他の臓器で発現するmRNAを標的とするsiRNA治療薬も開発中です。さらに、15のsiRNA治療薬が第2相および第3相臨床試験に進んでいます。第3相試験段階にある可能性のあるsiRNA治療薬には、それぞれLDH、APOC3、SERPINC1、TTRを標的とするネドシラン（DCR-PHXC）、ARO-APOC3、fitusiran（ALN-AT3SC）、revusiran（ALN-TTRSC）が含まれます。

RNAi治療薬の合併症と高コスト

有効性、毒性、特に肝臓への優先的蓄積による肝毒性、特異性／好ましい生体内分布、オフターゲット蓄積、長期安全性、免疫原性-siRNA分子とその生物学的作用に関する問題（オフターゲット効果、免疫原性、長期安全性、オフターゲット蓄積／RNA誘導サイレンシング複合体の飽和）。もう一つの大きな問題は、薬剤費が非常に高いことです。オンパトロの現在の米国での薬価は年間約45万米ドルですが、これは高い開発費と患者数が比較的少ない（米国では約3,000人のhATTR患者）ことが要因です。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • 分子診断（がん）向け用途の拡大
    • 医薬品承認の増加
  • 抑制要因
    • RNAi治療薬に関連する合併症と高コスト
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• サプライチェーン分析
• 価格分析
• 規制分析
• ロシア・ウクライナ戦争の影響分析
• DMIの見解

第6章 COVID-19分析

第7章 タイプ別

• マイクロRNA
• 低分子干渉RNA
• その他

第8章 用途別

• 創薬・医薬品開発
• 治療
  • 腫瘍
  • 肝臓疾患
  • 神経疾患
  • 呼吸器疾患
  • 自己免疫疾患
  • 眼病
  • その他
• ドラッグデリバリー
• 農業
• その他

第9章 エンドユーザー別

• 研究・学術研究所
• 診断研究所
• その他

第10章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • イタリア
  • その他欧州
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 中国
  • インド
  • 日本
  • オーストラリア
  • その他アジア太平洋
• 中東・アフリカ

第11章 競合情勢

• 競合シナリオ
• 市況/シェア分析
• M&A分析

第12章 企業プロファイル

• Alnylam Pharmaceuticals, Inc.
  • 企業概要
  • 製品ポートフォリオと概要
  • 財務概要
  • 主な動向
• Ionis Pharmaceuticals, Inc.
• Atalanta Therapeutics
• Phio Pharmaceuticals
• Benitec Biopharma
• Novartis AG
• AstraZeneca
• Silence Therapeutics
• Arbutus Biopharma
• Sylentis S.A.

第13章 付録

Overview

Global RNAi Technology Market reached US$ 1.6 billion in 2022 and is expected to reach US$ 3.6 billion by 2030 growing with a CAGR of 10.3% during the forecast period 2023-2030.

Small nucleic acid fragments are employed in RNA interference (RNAi), a technology, to post-transcriptionally silence genes and treat cancer and other disorders. Through the use of this technology, proteins that lack the enzyme pocket required for binding can be made more target-specific.

By using this method, the target genes may be verified and important illness genes can be functionally evaluated, resulting in the creation of potent medicines. Therapeutics based on RNA interference (RNAi) offer a powerful method for rapidly identifying specific and potent inhibitors of disease targets from all molecular classes.

Furthermore, the increased demand for precise gene silencing across a range of applications, including drug development and agricultural biotechnology, expansion of research in these fields is driving up the RNAi technology market size.

Dynamics

Increasing Applications in Molecular Diagnostics (Cancer)

The rise in genetic and metabolic problems, the rise in molecular diagnostics applications, the prevalence of cancer, and the expansion of research and development for drug discovery are some of the factors driving the worldwide RNAi technology market growth. The benefits of RNAi in cancer therapy include efficient growth control of tumors in advanced stages, low cost, and excellent specificity.

Drug resistance might be decreased by using RNAi to simultaneously block several genes in different pathways. For instance, researchers discovered that by inhibiting SH3GL1 with siRNA, the EGFR/ERK/AP-1 pathway might reduce P-glycoprotein expression and reverse MDR. As more efficient delivery technologies are created, RNAi may also be employed to provide tailored medications as chemotherapy adjuvants for particular patients.

Furthermore, metal oxide nanomaterials such as manganese oxide, iron oxide, silica, and metal organic frameworks are also productively used for gene therapy using siRNA. Despite the siRNA carrier capacity, metal oxide nanomaterials have phototherapy and imaging potential due to their magnetic properties, which enable them to be good bioimaging tools for magnetic resonance imaging and combination therapy for theranostics applications.

Rising Drug Approvals

RNA interference mediated by small interfering RNAs (siRNAs) has been exploited to develop therapeutics. siRNAs can be a powerful therapeutic tool because the working mechanisms of siRNAs are straightforward. siRNAs have the advantages of higher specificity than chemical drugs and a high degree of safety. siRNAs are also suitable in terms of efficiency, and candidate groups of siRNA medicines can be developed quickly and easily

As of December 2022, five siRNA drugs have been approved by the US FDA. Patisiran (Onpattro), Givosiran (Givlaari), Lumasiran (Oxlumo), Inclisiran (Leqvio), and Vutrisiran (Amvutta). All five approved siRNA drugs target mRNAs expressed in the liver. This is unsurprising because siRNAs delivered into an animal rapidly accumulate in the liver, a significant organ for detoxifying exogenous materials. However, siRNA drugs targeting mRNAs expressed in other organs are also under development. Moreover, fifteen siRNA therapeutics have progressed to phase 2 and 3 clinical trials. The potential siRNA drugs in phase 3 include nedosiran (DCR-PHXC), ARO-APOC3, fitusiran (ALN-AT3SC), and revusiran (ALN-TTRSC), which target LDH, APOC3, SERPINC1, and TTR, respectively.

Complications related to RNAi Therapeutics and High Costs

Efficacy, toxicity, especially hepatotoxicity due to preferential accumulation in the liver, specificity/favorable biodistribution, off-target accumulation, long-term safety, immunogenicity - as well as issues related to siRNA molecule and its biological actions (off-target effects, immunogenicity, long-term safety, off-target accumulation/RNA-induced silencing complex saturation). Another major issue may be very high drug costs. The current US price tag of Onpattro is approximately US$450,000 per year, which is explained by high development costs and comparably small patient numbers (-3000 hATTR patients in the USA)

Segment Analysis

The global RNAi technology market is segmented based on type, application, end-user and region.

The Therapeutics Segment Accounted for Approximately 38.2% of the Market Share

Due to the rise in demand for cancer and liver problems, therapeutics is the most rapidly expanding industry. Further, subsectors of the therapeutics market include cancer, liver diseases, neurological disorders, autoimmune disorders, respiratory disorders, ophthalmic disorders, etc.

Due to the increasing demand for better diagnostic and therapeutic approaches for the rising number of cancer cases, the oncology segment held the greatest market share in the global RNAi technology market. The World Health Organization in 2023, estimates that cancer accounts for 8.8 million deaths annually, making it the second largest cause of death worldwide. Globally, cancer is responsible for around one out of every six fatalities.

Over the following two decades, there will likely be a 70% increase in the number of new cases. This barrier can be removed in the clinical use of RNAi-based treatments for the treatment of cancer thanks to recent developments such as the creation of small interfering RNA (siRNA) resistant to nucleases and the discovery of non-viral vectors, including cationic liposomes and nanoparticles. To give accurate treatment, this technology offers a focused approach to cancer therapy. Additionally, it is predicted that the market will have future growth prospects thanks to the expanding medication pipeline, therapy approvals, and clinical trials.

Geographical Analysis

North America accounted for Approximately 39.3% of the Market Share in 2022

Due to the rising need for RNAi technology for treatment purpose in healthcare, manufacturers in North America have chances of increasing their operations. Increasing application of RNAi Technology in the healthcare industry and rising awareness among people, advancement of technologies for treatment, and increase in biopharmaceutical establishment across the region are also contributing to the growth of the RNAi technology market share of this region.

on April 25, 2023, Ionis Pharmaceuticals, Inc. and its partner Biogen has received U.S. Food and Drug Administration (FDA) approval of QALSODY (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.

With four siRNA medications already approved by the US Food and Drug Administration (FDA), several RNAi-based therapeutics continue to advance to clinical trials with functions that closely resemble their endogenous counterparts. It aids in enhancing stability and improving the efficacy of the therapeutics which impacts on the treatment outcome.

COVID-19 Impact Analysis

The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global RNAi technology market, as countries grappled with lockdowns, supply chain disruptions, and reduced economic activity.

The onset of the pandemic in early 2020 led to widespread lockdowns and restrictions, impacting drug development activities worldwide. The COVID-19 pandemic severely disrupted global supply chains, affecting the transportation of raw materials to pharmaceutical manufacturing industries.

Due to an unexpected halt in the research, the gene silencing technology did not move forward in innovations and focused on COVID-19 treatment. During the recent pandemic caused by SARS-CoV-2, mRNA-based vaccination strategies have paved the way for a new era of RNA therapeutics. RNA Interference (RNAi) based approach using small interfering RNA may complement the clinical management of COVID-19.

By Type

• Micro RNA
• Small interfering RNA
• Others

By Application

• Drug Discovery and Development
• Therapeutics
  • Oncology
  • Liver Disorders
  • Neurological Disorders
  • Respiratory Disorders
  • Autoimmune Disorders
  • Ocular Disorders
  • Others

• Others

By End User

• Research and Academic Laboratories
• Diagnostic Laboratories
• Others

By Region

• North America
  • U.S.
  • Canada
  • Mexico

• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe

• South America
  • Brazil
  • Argentina
  • Rest of South America

• Asia-Pacific
  • China
  • India
  • Japan
  • Australia
  • Rest of Asia-Pacific

• Middle East and Africa

Key Developments

• On May 18, 2023, pre-clinical data showing that employing INTASYL for silencing CBL-B and TIGIT may be utilized to enhance the anti-tumor response of NK cells, developing a more efficient cell therapy for managing cancer, were released by Phio Pharmaceuticals Corp., a development stage biotechnology business with exclusive INTASYL RNAi platform technology has been developed to enhance the effectiveness of immune cells in killing tumor cells.
• On April 26, 2023, Aln-APP, an investigational RNAi therapy that targets amyloid precursor protein (APP), was developed for the treatment of cerebral amyloid angiopathy (CAA) and Alzheimer's disease, and Regeneron Pharmaceuticals, Inc. and Alnylam Pharmaceuticals, Inc. recently announced encouraging preliminary outcomes from the in-progress single ascending dose part of ALN-APP Phase 1 study.
• On January 5, 2023, a master service agreement was signed between Orbit Identification Limited, a pioneer in the identification of therapeutic peptide hits, and SanegeneBio Inc. (Sanegene), a start-up focused on creating new RNAi-based medications. The goal of the partnership is to find effective ways to deliver a variety of RNA therapies to specific tissues in order to effectively knock off disease-causing genes.

Competitive Landscape

The major global players in the RNAi technology market include: Alnylam Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., Atalanta Therapeutics, Phio Pharmaceuticals, Benitec Biopharma, Novartis AG, AstraZeneca, Silence Therapeutics, Arbutus Biopharma, and Sylentis S.A. among others.

Why Purchase the Report?

• To visualize the global RNAi technology market segmentation based on type, application, end-user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of RNAi technology market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global RNAi technology market report would provide approximately 53 tables, 54 figures, and 195 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Type
• 3.2. Snippet by Application
• 3.3. Snippet by End User
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Increasing Applications in Molecular Diagnostics (Cancer)
    • 4.1.1.2. Rising Drug Approvals
  • 4.1.2. Restraints
    • 4.1.2.1. Complications related to RNAi Therapeutics and High Costs
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis
• 5.5. Russia-Ukraine War Impact Analysis
• 5.6. DMI Opinion

6. COVID-19 Analysis

• 6.1. Analysis of COVID-19
  • 6.1.1. Scenario Before COVID
  • 6.1.2. Scenario During COVID
  • 6.1.3. Scenario Post COVID
• 6.2. Pricing Dynamics Amid COVID-19
• 6.3. Demand-Supply Spectrum
• 6.4. Government Initiatives Related to the Market During Pandemic
• 6.5. Manufacturers Strategic Initiatives
• 6.6. Conclusion

7. By Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 7.1.2. Market Attractiveness Index, By Type
• 7.2. Micro RNA
• 7.3. Small interfering RNA
• 7.4. Others

8. By Application

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 8.1.2. Market Attractiveness Index, By Application
• 8.2. Drug Discovery and Development
• 8.3. Therapeutics
  • 8.3.1. Introduction
  • 8.3.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.3.3. Oncology
  • 8.3.4. Liver Disorders
  • 8.3.5. Neurological Disorders
  • 8.3.6. Respiratory Disorders
  • 8.3.7. Autoimmune Disorders
  • 8.3.8. Ocular Disorders
  • 8.3.9. Others
• 8.4. Drug Delivery
• 8.5. Agriculture
• 8.6. Others

9. By End User

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 9.1.2. Market Attractiveness Index, By End User
• 9.2. Research and Academic Laboratories
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Diagnostic Laboratories
• 9.4. Others

10. By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 10.1.2. Market Attractiveness Index, By Region
• 10.2. North America
  • 10.2.1. Introduction
  • 10.2.2. Key Region-Specific Dynamics
  • 10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.2.6.1. U.S.
    • 10.2.6.2. Canada
    • 10.2.6.3. Mexico
• 10.3. Europe
  • 10.3.1. Introduction
  • 10.3.2. Key Region-Specific Dynamics
  • 10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
  • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.3.6.1. Germany
    • 10.3.6.2. UK
    • 10.3.6.3. France
    • 10.3.6.4. Italy
    • 10.3.6.5. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.4.5.1. Brazil
    • 10.4.5.2. Argentina
    • 10.4.5.3. Rest of South America
• 10.5. Asia-Pacific
  • 10.5.1. Introduction
  • 10.5.2. Key Region-Specific Dynamics
  • 10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.5.5.1. China
    • 10.5.5.2. India
    • 10.5.5.3. Japan
    • 10.5.5.4. Australia
    • 10.5.5.5. Rest of Asia-Pacific
• 10.6. Middle East and Africa
  • 10.6.1. Introduction
  • 10.6.2. Key Region-Specific Dynamics
  • 10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Market Positioning/Share Analysis
• 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

• 12.1. Alnylam Pharmaceuticals, Inc.
  • 12.1.1. Company Overview
  • 12.1.2. Type Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Ionis Pharmaceuticals, Inc.
• 12.3. Atalanta Therapeutics
• 12.4. Phio Pharmaceuticals
• 12.5. Benitec Biopharma
• 12.6. Novartis AG
• 12.7. AstraZeneca
• 12.8. Silence Therapeutics
• 12.9. Arbutus Biopharma
• 12.10. Sylentis S.A.

LIST NOT EXHAUSTIVE

13. Appendix

• 13.1. About Us and Services
• 13.2. Contact Us