過去2年間、核酸ベースの治療法への需要が増加しました。これは、COVID-19の治療法を開発するために効果的に使用されたためです。2022年2月にMDPIが発表した記事によると、核酸ベースの技術（小干渉RNA（siRNA）、アンチセンスオリゴヌクレオチド（ASO）、マイクロRNA（miRNA）を含む）は、COVID-19ウイルスと戦うための最も有望なものでした。これらの治療法は、転写中および転写後にウイルス遺伝子発現を抑制することができ、重要な成長機会を提供します。2022年8月にMDPIが発表した記事によると、世界中で400件以上のRNA標的薬物開発プロジェクトが実施され、その3分の2は前研究段階（pre-IND）で、3分の1は早期臨床試験（フェーズIまたはII）にあり、約3％がフェーズIIIにあり、一部はCOVID-19治療のための規制承認を待っています。このように、RNA治療法の開発が進む中で、調査対象市場は予測期間中に成長することが期待されています。

• 1 イントロダクション

  • 1.1 調査の前提条件と市場の定義

  • 1.2 本調査の範囲

• 2 調査手法

• 3 エグゼクティブサマリー

• 4 市場ダイナミクス

  • 4.1 市場概要

  • 4.2 市場の促進要因

    • 4.2.1 遺伝性疾患の急増

    • 4.2.2 ヘルスケア分野への投資拡大

    • 4.2.3 革新的生物製剤への製薬業界の急速なシフト

  • 4.3 市場の抑制要因

    • 4.3.1 核酸研究の高いコスト

  • 4.4 ポーターのファイブフォース分析

    • 4.4.1 新規参入の脅威

    • 4.4.2 買い手・消費者の交渉力

    • 4.4.3 サプライヤーの交渉力

    • 4.4.4 代替品の脅威

    • 4.4.5 競合・競争状況の激しさ

• 5 市場セグメンテーション（市場規模別、金額、百万米ドル）

  • 5.1 製品タイプ別

    • 5.1.1 RNA干渉[RNAi]と短鎖干渉RNA[siRNA]

    • 5.1.2 アンチセンスオリゴヌクレオチド（ASOs）

    • 5.1.3 その他の製品タイプ

  • 5.2 用途別

    • 5.2.1 自己免疫疾患

    • 5.2.2 感染症

    • 5.2.3 遺伝性疾患

    • 5.2.4 がん

    • 5.2.5 その他の用途

  • 5.3 エンドユーザー別

    • 5.3.1 病院・クリニック

    • 5.3.2 アカデミック・研究機関

  • 5.4 地域別

    • 5.4.1 北米

      • 5.4.1.1 米国
      • 5.4.1.2 カナダ
      • 5.4.1.3 メキシコ

    • 5.4.2 ヨーロッパ

      • 5.4.2.1 ドイツ
      • 5.4.2.2 英国
      • 5.4.2.3 フランス
      • 5.4.2.4 イタリア
      • 5.4.2.5 スペイン
      • 5.4.2.6 その他のヨーロッパ

    • 5.4.3 アジア太平洋

      • 5.4.3.1 中国
      • 5.4.3.2 日本
      • 5.4.3.3 インド
      • 5.4.3.4 オーストラリア
      • 5.4.3.5 韓国
      • 5.4.3.6 その他のアジア太平洋

    • 5.4.4 中東・アフリカ

      • 5.4.4.1 GCC地域
      • 5.4.4.2 南アフリカ
      • 5.4.4.3 その他の中東・アフリカ

    • 5.4.5 南米

      • 5.4.5.1 ブラジル
      • 5.4.5.2 アルゼンチン
      • 5.4.5.3 その他の南米

• 6 競合情勢

  • 6.1 企業プロファイル

    • 6.1.1 Silence Therapeutics PLC

    • 6.1.2 Ionis Pharmaceuticals Inc

    • 6.1.3 Novartis Pharma AG

    • 6.1.4 Arrowhead Pharmaceuticals Inc

    • 6.1.5 Stoke Therapeutics Inc

    • 6.1.6 Moderna Inc

    • 6.1.7 Alnylam Pharmaceuticals Inc

    • 6.1.8 Biogen Inc

    • 6.1.9 Wave Life Sciences

    • 6.1.10 Sarepta Therapeutics Inc

• 7 市場機会と今後の動向

The Nucleic Acid Based Therapeutics Market size is estimated at USD 5.59 billion in 2024, and is expected to reach USD 10.90 billion by 2029, growing at a CAGR of 14.29% during the forecast period (2024-2029).

The demand for nucleic acid-based therapeutics increased over the past two years as they were effectively used to develop COVID-19 disease therapeutics. As per the article published by MDPI in February 2022, nucleic acid-based technologies, including small interfering RNAs (siRNAs), antisense oligonucleotides (ASOs), and micro RNAs (miRNAs), were the most promising ones to combat the COVID-19 virus. These therapeutics can suppress viral gene expression during and after transcription, presenting significant growth opportunities. As per an article published in MDPI, in August 2022, over 400 RNA-targeting drug development projects were conducted globally, two-thirds of which are in the pre-investigational new drug (pre-IND) stage, one-third in early clinical trials (Phase I or II), about 3% in Phase III, and some awaiting regulatory approval for COVID-19 treatment. Thus, with the increasing development of RNA therapeutics, the studied market is expected to grow over the forecast period.

Factors such as the surging prevalence of genetic disease, growing investments in the healthcare sector and rapid shift of the pharmaceutical industry toward innovative biologics are expected to boost the market's growth over the forecast period.

The increasing prevalence of genetic diseases such as cancer, cystic fibrosis, sickle cell anemia, Duchenne muscular dystrophy, thalassemia, and others is the key factor driving the demand for nucleic acid-based therapeutics to treat diseases by correcting the abnormal expression of specific genes. For instance, according to the Cystic Fibrosis Foundation data published in July 2022, in the United States, 40,000 children and adults have cystic fibrosis (CF). As per the same source, an estimated 105,000 people were diagnosed with the disease in 94 countries in 2022. Thus, the high burden of cystic fibrosis among the population is expected to positively impact the market.

The rising number of investments in nucleic acid therapeutics owing to the high prevalence of chronic, genetic, and other diseases is anticipated to augment the market's growth. For instance, in March 2023, Switch Therapeutics raised USD 52 million in a Series A financing round co-led by Insight Partners and UCB Ventures. The company used these funds to select and advance the development of siRNA therapeutic candidates for the treatment of a central nervous system disease, as well as to advance its RNAi technology. Market players are investing in nucleic acid-based therapy R&D to expand the product portfolio in nucleic acid-based therapy. For instance, in January 2023, Agilent Technologies Inc. invested USD 725 million in nucleic acid-based therapeutics. The investment will double the manufacturing capacity to produce active pharmaceutical ingredients (APIs).

Companies' growing focus on collaborations, partnerships, new product launches, and other initiatives increases the availability of novel therapeutic drugs in the market, which may propel the market's growth. For instance, in February 2023, the CMT Research Foundation partnered with Nanite Inc. to enhance the therapeutic efficacy of antisense oligonucleotides in Charcot-Marie-Tooth disease. In October 2022, Neuway Pharma GmbH and Wacker launched a research project to identify and manufacture RNA-based therapeutics for the treatment of central nervous system disorders with the use of EnPC (Engineered Protein Capsules) protein-based drug delivery technology.

Therefore, owing to the high burden of genetic diseases, increasing investments, and new product launches, the market studied is anticipated to grow over the forecast period. However, the high cost of nucleic acid-based therapeutics research is likely to impede the market's growth.

Nucleic Acid Based Therapeutics Market Trends

Antisense Oligonucleotides (ASOs) Segment is Expected to Witness Significant Growth Over the Forecast Period

Antisense oligonucleotides (ASOs) are short, single-stranded DNA or RNA molecules that can bind to specific messenger RNA (mRNA) sequences, leading to the degradation of the targeted mRNA or inhibition of its translation into protein. This property makes ASOs a promising therapeutic strategy for a variety of diseases, including genetic disorders, infectious diseases, and cancer. These can target specific disease-causing genes, such as those responsible for the production of abnormal proteins or enzymes that contribute to disease progression.

Some examples of approved ASO therapies include Spinraza (nusinersen) for the treatment of spinal muscular atrophy, Exondys51 (eteplirsen) for the treatment of Duchenne muscular dystrophy, Onpattro (patisiran) for the treatment of hereditary transthyretin-mediated amyloidosis, and Tegsedi (inotersen) for the treatment of hereditary transthyretin-mediated amyloidosis.

The ASOs segment is expected to witness significant growth over the forecast period due to the high demand for antisense oligonucleotide drugs, increasing R&D activities by key players, and new product launches.

The increasing focus of the companies to adopt various business strategies such as partnerships, collaborations, acquisitions, and other initiatives to accelerate the R&D of novel therapeutics for rare and genetic disorders is anticipated to fuel the segment's growth. For instance, in September 2022, Vanda Pharmaceuticals Inc. and OliPassCorporation entered an R&D collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on modified peptide nucleic acids. In February 2021, the US FDA approved Sarepta's Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene.

Therefore, the segment is expected to grow over the forecast period due to the availability of several antisense oligonucleotide products in the market, increasing company activities, and new product launches.

North America is Expected to Have the Significant Market Share Over the Forecast Period

North America is expected to hold a significant share of the market due to the growing research on various nucleic acid-based therapies, the increasing prevalence of genetic disorders and other chronic disorders, and growing R&D investments in the region.

The rising prevalence of autoimmune diseases among the population increases the demand for nucleic acid-based therapies, which may augment the market's growth. For instance, as per the Autoimmune Association in June 2022, autoimmune diseases comprise approximately 80-150 unique, chronic conditions and affect more than 31 million Americans annually. This shows the high burden of autoimmune disorders among the target population. As per an article published in the Clinical Rheumatology Journal in February 2023, the burden of systemic autoimmune rheumatic diseases (SARDs) is large in Canada, affecting between 2 and 5 cases per 1,000 residents.

The increasing burden of cancer raises the demand for effective and novel drugs that inhibit the progression of cancer cells. This fuels the demand for nucleic acid-based drugs in the region. For instance, according to the Cancer Facts and Figures 2023, about 1.9 million new cancer cases are estimated to be diagnosed in the country in 2023. According to the statistics updated by the Government of Canada in June 2022, nearly 233,900 people in Canada were estimated to be diagnosed with cancer by the end of 2022, and lung, breast, prostate, and colorectal cancers were predicted to be the most diagnosed cancers among the target population in Canada.

The innovative product launches and approvals, partnerships, acquisitions, expansions, and collaborations are anticipated to fuel the market's growth in the region. For instance, in September 2022, Next Generation Manufacturing Canada (NGen) invested USD 10.5 million in a USD 34.8 million project led by OmniaBio Inc. and partners ExCellThera, MorphoCell Technologies, Aspect Biosystems, and Canadian Advanced Therapies Training Institute (CATTI). In December 2021, Novartis AG received approval from the FDA for Leqvio (inclisiran), the first and only small interfering RNA (siRNA) therapy to lower low-density lipoprotein cholesterol (also known as bad cholesterol or LDL-C) with two doses a year, after an initial dose and one at three months.

Nucleic Acid Based Therapeutics Industry Overview

The nucleic acid-based therapeutics market is moderately fragmented due to the presence of many small and large players globally and regionally. The companies are engaging in the research and development of nucleic acid-based therapeutics for the treatment of various chronic, genetic, and infectious diseases. Some of the key companies in the market are Silence Therapeutics PLC, Ionis Pharmaceuticals Inc., Sarepta Therapeutics, Novartis Pharma AG, Alnylam Pharmaceuticals Inc., and Biogen Inc., among others.

Additional Benefits:

• The market estimate (ME) sheet in Excel format
• 3 months of analyst support

• 1 INTRODUCTION

  • 1.1 Study Assumptions and Market Definition

  • 1.2 Scope of the Study

• 2 RESEARCH METHODOLOGY

• 3 EXECUTIVE SUMMARY

• 4 MARKET DYNAMICS

  • 4.1 Market Overview

  • 4.2 Market Drivers

    • 4.2.1 Surging Prevalence of Genetic Diseases

    • 4.2.2 Growing Investments in Healthcare Sector

    • 4.2.3 Rapid Shift of the Pharmaceutical Industry Toward Innovative Biologics

  • 4.3 Market Restraints

    • 4.3.1 High Cost of Nucleic Acid Research

  • 4.4 Porter's Five Forces Analysis

    • 4.4.1 Threat of New Entrants

    • 4.4.2 Bargaining Power of Buyers/Consumers

    • 4.4.3 Bargaining Power of Suppliers

    • 4.4.4 Threat of Substitute Products

    • 4.4.5 Intensity of Competitive Rivalry

• 5 MARKET SEGMENTATION (Market Size by Value in USD million)

  • 5.1 By Product Type

    • 5.1.1 RNA interference [RNAi] and short interfering RNAs [siRNAs]

    • 5.1.2 Antisense Oligonucleotides (ASOs)

    • 5.1.3 Other Product Types

  • 5.2 By Application

    • 5.2.1 Autoimmune Disorders

    • 5.2.2 Infectious Diseases

    • 5.2.3 Genetic Disorders

    • 5.2.4 Cancer

    • 5.2.5 Other Applications

  • 5.3 By End User

    • 5.3.1 Hospitals and Clinics

    • 5.3.2 Academic and Research Institutes

  • 5.4 By Geography

    • 5.4.1 North America

      • 5.4.1.1 United States
      • 5.4.1.2 Canada
      • 5.4.1.3 Mexico

    • 5.4.2 Europe

      • 5.4.2.1 Germany
      • 5.4.2.2 United Kingdom
      • 5.4.2.3 France
      • 5.4.2.4 Italy
      • 5.4.2.5 Spain
      • 5.4.2.6 Rest of Europe

    • 5.4.3 Asia-Pacific

      • 5.4.3.1 China
      • 5.4.3.2 Japan
      • 5.4.3.3 India
      • 5.4.3.4 Australia
      • 5.4.3.5 South Korea
      • 5.4.3.6 Rest of Asia-Pacific

    • 5.4.4 Middle East & Africa

      • 5.4.4.1 GCC
      • 5.4.4.2 South Africa
      • 5.4.4.3 Rest of Middle East & Africa

    • 5.4.5 South America

      • 5.4.5.1 Brazil
      • 5.4.5.2 Argentina
      • 5.4.5.3 Rest of South America

• 6 COMPETITIVE LANDSCAPE

  • 6.1 Company Profiles

    • 6.1.1 Silence Therapeutics PLC

    • 6.1.2 Ionis Pharmaceuticals Inc

    • 6.1.3 Novartis Pharma AG

    • 6.1.4 Arrowhead Pharmaceuticals Inc

    • 6.1.5 Stoke Therapeutics Inc

    • 6.1.6 Moderna Inc

    • 6.1.7 Alnylam Pharmaceuticals Inc

    • 6.1.8 Biogen Inc

    • 6.1.9 Wave Life Sciences

    • 6.1.10 Sarepta Therapeutics Inc

• 7 MARKET OPPORTUNITIES AND FUTURE TRENDS