Antisense & RNAi Therapeutics Market Size & Share Analysis - Growth Trends & Forecasts (2024 - 2029)

The Global Antisense & RNAi Therapeutics Market is segmented by Therapeutics (RNA Interference and RNA Antisense), Route of Administration (Intravenous Route, Subcutaneous Route, Intrathecal Route, Pulmonary Delivery, Intraperitoneal Injection, and Others), Indication (Autosomal Recessive Disease, Autosomal Dominant Disease, Chromosomal Disease, and Others), and Geography (North America, Europe, Asia-Pacific, Middle East, and Africa, and South America). The report offers the value (in USD million) for the above segments.

Antisense & RNAi Therapeutics Market Size

Antisense and RNAi Therapeutics Market Summary
Study Period 2019 - 2029
Base Year For Estimation 2023
CAGR 7.10 %
Fastest Growing Market Asia-Pacific
Largest Market North America
Market Concentration High

Major Players

Antisense and RNAi Therapeutics Market Major Players

*Disclaimer: Major Players sorted in no particular order

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Antisense & RNAi Therapeutics Market Analysis

The antisense and RNAi therapeutics market is expected to register a CAGR of 7.1% over the forecast period, 2022-2027.

The COVID-19 pandemic led to seriously deleterious effects on public health systems in many countries. For instance, the healthcare industry was burdened with pandemic crisis management. Furthermore, worldwide lockdowns were imposed to curb the viral outbreak. This in turn led to a shortage of healthcare staff across the globe. Similarly, the pharmaceuticals' manufacturing activities and supply chain worldwide. were also obstructed. In addition, the Antisense and RNAi therapeutics market also suffered substantial losses during the COVID-19 pandemic. There was a sharp decline in the outpatient services offered in hospitals and clinics during the pandemic. Thus, patients receiving treatment for rare genetic conditions such as spinal muscular atrophy (SMA), acute hepatic porphyria, Duchenne muscular dystrophy (DMD), and others experienced delayed drug infusion or disruptions in drug infusion services provided by the healthcare staff. Hence, this leads to a decrease in demand for antisense and RNAi therapeutics. For instance, according to a survey finding released by National Organization for Rare Disorders, Inc. (NORD) in May 2020 in the United States, 74% of patients suffering from rare diseases had a medical appointment canceled. Similarly, according to a survey conducted by the Rare Diseases Clinical Research Network (RDCRN) in the year 2020, only a few patients suffering from rare diseases were able to get an appointment with a medical provider. Thus, the decline in hospital visits during the pandemic impacted the growth of the studied market.

The major factor that leads to the growth of the studied market includes better therapeutic outcomes for rare diseases with the use of antisense and RNAi therapeutics. Furthermore, other factors such as the surge in R&D funding coupled with the presence of promising pipeline products also drive the growth of the market.

RNAi therapeutics are based on a technology that works by preventing cells from producing a specific protein that contributes to disease. Furthermore, this technology is also called gene silencing technology. Some of these rare diseases include spinal muscular atrophy, heterozygous familial hypercholesterolemia (HeFH), acute hepatic porphyria, and others. Hence, this technique has improved the treatment options for rare diseases. For instance, the siRNA therapies identify and bind to the disease-causing mRNA leading to its destruction. Hence, this renders RNAi therapeutics as highly target specific and selective making them a preferred and advanced treatment option for rare diseases. Thus, this leads to a surge in demand for RNAi therapeutics, thereby driving the growth of the market.

In addition, the treatment options available for rare disease across the world is limited. Hence, there has been a gradual increase in the funding related to research activities associated with the discovery of new therapies for rare diseases. For instance, in January 2022, the Food and Drug Administration Office of Orphan Products Development (OOPD) announced the availability of funds to support studies for rare diseases and conditions. In addition, these studies are also expected to provide data to the FDA contributing to the approval of new products, or new indications for already marketed products. Hence, this is expected to lead to a surge in R&D activities related to antisense and RNAi therapeutics for rare diseases. Furthermore, the presence of strong pipeline products which are expected to be launched during the forecast period also drives the growth of the market. For instance, AB-729 is an RNAi therapeutic candidate which is currently being studied by Arbutus Biopharma. Furthermore, AB-729 is in Phase II of clinical trials and is being studied for its therapeutic action in Hepatitis B patients. Similarly, ISTH0036 is another RNAi therapeutic being studied by Isarna Therapeutics GmbH. The drug candidate is present in phase II of clinical trials and is being studied for diabetic macular edema. In addition, GS-101 (Aganirsen) is an antisense oligonucleotide studied by Gene Signal International SA. The drug candidate is currently in phase III of a clinical trial for keratoplasty rejection. Thus, these drug candidates are expected to hit the market in near future. Furthermore, this is expected to lead to a surge in demand for this novel RNAi and antisense therapeutics thereby driving the growth of the market.

Antisense & RNAi Therapeutics Market Trends

This section covers the major market trends shaping the Antisense & RNAi Therapeutics Market according to our research experts:

RNA Antisense is Expected to Witness High Growth Over the Forecast Period

RNA antisense therapeutics are widely used in the treatment of rare genetic disorders such as spinal muscular atrophy, Duchenne muscular dystrophy (DMD), and hereditary transthyretin amyloidosis (hATTR). Furthermore, the factors that contribute to the growth of this market include a surge in awareness related to the treatment of these rare genetic diseases. Thus, the surge in awareness leads to a rise in demand for RNA antisense therapeutics.

Similarly, factors such as the high cost of RNA antisense therapies such as Spinraza offered by Biogen Inc. and Exondys 51 offered by Sarepta Therapeutics, Inc. and lead to a surge in revenues in the market. For instance, according to a report published by Institute for Clinical and Economic Review (ICER) in 2109, Spinraza's price for patients with presymptomatic spinal muscular atrophy is between $72,000-$130,000 for the first year of treatment. Furthermore, recent product approvals in the market lead to a surge in demand for novel and advanced RNA antisense therapeutics thereby driving the growth of the market. For instance, in February 2021, Sarepta Therapeutics, Inc. received US Food and Drug Administration (US FDA) approval for AMONDYS 45 (casimersen), an antisense oligonucleotide. Furthermore, AMONDYS 45 is indicated for the treatment of DMD. Hence, the factors mentioned above contribute to the growth of the RNA antisense market.

Antisense and RNAi Therapeutics Market : Number of Patent Appications for RNAi Therapeutics, 2019-2021

North America is Expected to Dominate the Antisense and RNAi Therapeutics Market

North America is expected to dominate the market. This is due to the high usage of antisense and RNAi therapeutics such as Spinraza offered by Biogen Inc., Onpattro and Givlaari offered by Alnylam Pharmaceuticals, Inc., and others in the region. In addition, other factors that contribute to the growth of the market in the region include the availability of advanced antisense and RNAi therapeutics. Furthermore, the presence of well-established healthcare infrastructure in the region leads to a surge in early screening of rare diseases which in turn leads to a surge in demand for antisense and RNAi therapeutics. For instance, Cure SMA is a non-profit organization dedicated to promoting and supporting research related to spinal muscular atrophy. Furthermore, in June 2021, Cure SMA reported that 85% of newborns in the United States were screened for spinal muscular atrophy.

In addition, in August 2022, Cure SMA announced the addition of 9 new spinal muscular atrophy screening centers to its Care Center Network. In addition, currently, there are 27 such care centers in this network across the United States. Thus, this leads to a surge in early screening in the region which in turn leads to a surge in demand for antisense and RNAi therapeutics.

Therefore, owing to the previously mentioned factors the growth of the market studied is anticipated in the North America Region.

Antisense and RNAi therapeutics market Growth Rate By Region

Antisense & RNAi Therapeutics Industry Overview

The antisense and RNAi therapeutics market is consolidated in nature due to the presence of a limited number of companies operating across the globe. The competitive landscape includes an analysis of a few international as well as local companies that operate in the market such as Alnylam Pharmaceuticals, Inc., Biogen Inc., Ionis Pharmaceuticals (Akcea Therapeutics, Inc.), Sarepta Therapeutics, Inc., Novartis AG, Silence Therapeutics, Arbutus Biopharma (Formerly Called Tekmira Pharmaceuticals), Isarna Therapeutics GmbH, Gene Signal International SA, Arrowhead Pharmaceuticals, Inc., and Benitec Biopharma Ltd, among others.

Antisense & RNAi Therapeutics Market Leaders

  1. Alnylam Pharmaceuticals, Inc.

  2. Biogen Inc.

  3. Ionis Pharmaceuticals (Akcea Therapeutics, Inc.)

  4. Sarepta Therapeutics, Inc.

  5. Novartis AG

*Disclaimer: Major Players sorted in no particular order

Antisense and RNAi Therapeutics Market Concentration
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Antisense & RNAi Therapeutics Market News

  • In December 2021, Novartis AG received Food and Drug Administration (FDA) approval for Leqvio (inclisiran). Furthermore, Leqvio is a small interfering RNA (siRNA) therapy approved for lowering low-density lipoprotein cholesterol (LDL).
  • In December 2021, AstraZeneca plc and Ionis Pharmaceuticals entered into global development and commercialization agreement for eplontersen, an antisense investigational medicine. Furthermore, eplontersen is currently being studied in phase II of clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN).
  • In July 2021, Alnylam Pharmaceuticals, Inc. and PeptiDream Inc. entered into a licensing and collaboration agreement. Furthermore, the agreement is aimed at the discovery and development of peptide-siRNA conjugates to create multiple opportunities to deliver RNAi therapeutics to tissues outside the liver.

Antisense & RNAi Therapeutics Market Report - Table of Contents

  1. 1. INTRODUCTION

    1. 1.1 Study Assumptions and Market Definition

    2. 1.2 Scope of the Study

  2. 2. RESEARCH METHODOLOGY

  3. 3. EXECUTIVE SUMMARY

  4. 4. MARKET DYNAMICS

    1. 4.1 Market Overview

    2. 4.2 Market Drivers

      1. 4.2.1 Better Therapeutic Outcomes for Rare Disease Due to Effective Gene Slicing Technology

      2. 4.2.2 Surge in R&D funding Coupled with Presence of Promising Pipeline Products

    3. 4.3 Market Restraints

      1. 4.3.1 High Cost of Therapeutics

    4. 4.4 Porter's Five Force Analysis

      1. 4.4.1 Threat of New Entrants

      2. 4.4.2 Bargaining Power of Buyers/Consumers

      3. 4.4.3 Bargaining Power of Suppliers

      4. 4.4.4 Threat of Substitute Products

      5. 4.4.5 Intensity of Competitive Rivalry

  5. 5. MARKET SEGMENTATION (Market Size by Value - USD million)

    1. 5.1 By Therapeutics

      1. 5.1.1 RNA Interference

      2. 5.1.2 RNA Antisense

    2. 5.2 By Route of Administration

      1. 5.2.1 Intravenous Route

      2. 5.2.2 Subcutaneous Route

      3. 5.2.3 Intrathecal Route

      4. 5.2.4 Pulmonary Delivery

      5. 5.2.5 Intraperitoneal Injection

      6. 5.2.6 Others

    3. 5.3 By Indication

      1. 5.3.1 Autosomal Recessive Disease

      2. 5.3.2 Autosomal Dominant Disease

      3. 5.3.3 Chromosomal Disease

      4. 5.3.4 Others

    4. 5.4 Geography

      1. 5.4.1 North America

        1. 5.4.1.1 United States

        2. 5.4.1.2 Canada

        3. 5.4.1.3 Mexico

      2. 5.4.2 Europe

        1. 5.4.2.1 Germany

        2. 5.4.2.2 United Kingdom

        3. 5.4.2.3 France

        4. 5.4.2.4 Italy

        5. 5.4.2.5 Spain

        6. 5.4.2.6 Rest of Europe

      3. 5.4.3 Asia-Pacific

        1. 5.4.3.1 China

        2. 5.4.3.2 Japan

        3. 5.4.3.3 India

        4. 5.4.3.4 Australia

        5. 5.4.3.5 South Korea

        6. 5.4.3.6 Rest of Asia-Pacific

      4. 5.4.4 Middle East and Africa

        1. 5.4.4.1 GCC

        2. 5.4.4.2 South Africa

        3. 5.4.4.3 Rest of Middle East and Africa

      5. 5.4.5 South America

        1. 5.4.5.1 Brazil

        2. 5.4.5.2 Argentina

        3. 5.4.5.3 Rest of South America

  6. 6. COMPETITIVE LANDSCAPE

    1. 6.1 Company Profiles

      1. 6.1.1 Alnylam Pharmaceuticals, Inc.

      2. 6.1.2 Biogen Inc.

      3. 6.1.3 Ionis Pharmaceuticals (Akcea Therapeutics, Inc.)

      4. 6.1.4 Sarepta Therapeutics, Inc.

      5. 6.1.5 Silence Therapeutics

      6. 6.1.6 Arbutus Biopharma (Formerly Called Tekmira Pharmaceuticals)

      7. 6.1.7 Isarna Therapeutics GmbH

      8. 6.1.8 Gene Signal International SA

      9. 6.1.9 Arrowhead Pharmaceuticals, Inc.

      10. 6.1.10 Benitec Biopharma Ltd

    2. *List Not Exhaustive
  7. 7. MARKET OPPORTUNITIES AND FUTURE TRENDS

**Subject to Availability
**Competitive Landscape covers- Business Overview, Financials, Products and Strategies and Recent Developments
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Antisense & RNAi Therapeutics Industry Segmentation

As per the scope of the report, antisense and RNAi therapeutics are defined as medications that work by turning off the production of specific genes which are responsible for diseases or medical conditions in the body. The antisense and RNAi therapeutics market is segmented by Therapeutics (RNA Interference and RNA Antisense), Route of Administration (Intravenous Route, Subcutaneous Route, Intrathecal Route, Pulmonary Delivery, Intraperitoneal Injection, and Others), Indication (Autosomal Recessive Disease, Autosomal Dominant Disease, Chromosomal Disease, and Others), and Geography (North America, Europe, Asia-Pacific, Middle East, and Africa, and South America). The market report also covers the estimated market sizes and trends for 17 different countries across major regions, globally. The report offers the value (in USD million) for the above segments.

By Therapeutics
RNA Interference
RNA Antisense
By Route of Administration
Intravenous Route
Subcutaneous Route
Intrathecal Route
Pulmonary Delivery
Intraperitoneal Injection
Others
By Indication
Autosomal Recessive Disease
Autosomal Dominant Disease
Chromosomal Disease
Others
Geography
North America
United States
Canada
Mexico
Europe
Germany
United Kingdom
France
Italy
Spain
Rest of Europe
Asia-Pacific
China
Japan
India
Australia
South Korea
Rest of Asia-Pacific
Middle East and Africa
GCC
South Africa
Rest of Middle East and Africa
South America
Brazil
Argentina
Rest of South America
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Antisense & RNAi Therapeutics Market Research FAQs

The Global Antisense and RNAi Therapeutics Market is projected to register a CAGR of 7.10% during the forecast period (2024-2029)

Alnylam Pharmaceuticals, Inc., Biogen Inc., Ionis Pharmaceuticals (Akcea Therapeutics, Inc.), Sarepta Therapeutics, Inc. and Novartis AG are the major companies operating in the Global Antisense and RNAi Therapeutics Market.

Asia-Pacific is estimated to grow at the highest CAGR over the forecast period (2024-2029).

In 2024, the North America accounts for the largest market share in Global Antisense and RNAi Therapeutics Market.

The report covers the Global Antisense and RNAi Therapeutics Market historical market size for years: 2019, 2020, 2021, 2022 and 2023. The report also forecasts the Global Antisense and RNAi Therapeutics Market size for years: 2024, 2025, 2026, 2027, 2028 and 2029.

Antisense & RNAi Therapeutics Industry Report

Statistics for the 2024 Antisense & RNAi Therapeutics market share, size and revenue growth rate, created by Mordor Intelligenceā„¢ Industry Reports. Antisense & RNAi Therapeutics analysis includes a market forecast outlook 2029 and historical overview. Get a sample of this industry analysis as a free report PDF download.

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Antisense & RNAi Therapeutics Market Size & Share Analysis - Growth Trends & Forecasts (2024 - 2029)