Antisense & RNAi Therapeutics Market Report

Antisense & RNAi Therapeutics Market Analysis Report By Application (Oncology, Cardiovascular, Infectious Diseases), By Technology (siRNA, miRNA), By Route of Administration, And Segment Forecasts, 2019 - 2025

  • Published Date: Jan, 2019
  • Base Year for Estimate: 2017
  • Report ID: 978-1-68038-805-3
  • Format: Electronic (PDF)
  • Historical Data: 2014 - 2017
  • Number of Pages: 161

Industry Insights

The global antisense & RNAi therapeutics market size was estimated at USD 1.02 billion in 2017 and is expected to exhibit a CAGR of 7.5% during the forecast period. Antisense technology provides the opportunity to manipulate gene expression for the treatment of various diseases and this is expected to serve as a driving factor for R&D in this sector. This technology is used to study gene function for the purpose of disease management by regulating the expression of the disease-causing gene. Apart from this, R&D carried out to study the treatment of uncommon neurodegenerative diseases such as hereditary ATTR amyloidosis is anticipated to fuel market growth.

Antisense and RNAi therapeutics enable highly specific and effective gene silencing and as a result, a fair number of gene silencing drugs are in the late stages of development. The market is expected to witness steady growth during the forecast period owing to an increasing number of companies investing in the development of molecules through antisense technology.

Japan antisense & RNAi therapeutics market

Rising collaborations between pharmaceutical companies and contract research organizations working on drug delivery is expected to emerge as a positive trend for market growth. However, overcoming challenges such as unpredictable activity from molecules and other delivery-associated complications could hamper market development.

In January 2016, Ionis Pharmaceuticals (previously Isis Pharmaceuticals) announced the initiation of Phase-I study of ISIS-HBV-L Rx in collaboration with GSK Pharmaceuticals for treatment of Hepatitis B. Another collaboration by Ionis Pharmaceuticals with Akcea Therapeutics led to the approval of the world’s first antisense based molecule Tegsdi by the FDA and the European Commission in 2018. Several other collaborations are being undertaken by companies such as Biogen Inc.; Akcea Therapeutics; Ionis Pharmaceuticals; GlaxoSmithKline plc; Suzhou Ribo Life Science Co. Ltd.; Janssen Biotech Inc.; and AstraZeneca plc. The impact of technology is expected to increase dramatically with growing understanding of disease etiology, protein production, and genetics.

However, difficulty in delivering drugs to the desired site is a main challenge associated with the use of antisense and RNAi. Drug delivery to nervous tissues is one of the many problems as the drug has to pass the blood-brain barrier. Companies are coming up with solutions to overcome these hurdles. For instance, Ionis Pharmaceuticals developed a unique method of drug delivery to the brain by virtue of a catheter or tube, which is connected to the pump inserted in the chest.

Inevitable toxic effects is another roadblock in the usage of this technology. Determining the right dosage and vector for drug delivery of an antisense molecule is one of the most important steps to ensure safe administration of the molecule. Companies have also faced rejection from regulatory bodies in this regard. For instance, Akcea’s Waylivra, an antisense molecule to treat familial chylomicronemia syndrome, has been rejected by the FDA. These factors have compelled companies to push for better vectors, delivery solutions, and compositions.

Technology Insights

Antisense RNA has led the market in terms of revenue generation, accounting for a share of around 95.0% in 2017. This interest in antisense RNA can be owed to growing demand and increasing awareness in the usage of antisense oligonucleotides for treatment of neurodegenerative disorders. These molecules are being tested to prevent disease onset and halt disease progression.

Recent studies indicate that spinal muscular atrophy and amyotrophic lateral sclerosis have been showing promising results with the use of antisense technology. In addition to this, many antisense oligonucleotides are being tested for treatment of non-neurodegenerative disorders. These include antisense-mediated exon skipping for Duchenne muscular dystrophy.

Route of Administration Insights

The global antisense & RNAi therapeutics market can be segmented on the basis of route of administration into pulmonary delivery, intravenous injections, intradermal injections, intraperitoneal injections, topical delivery, and others. Route of administration is one of the most important aspects of any genetically modified molecule. The efficacy of these molecules varies based on the method of administration, vector used for the genetically modified molecule, and type of molecule for injection. Most studies employ attenuated viral particles or bacterial cells and are injected intravenously for the treatment.

Scientists are developing novel delivery systems such as nanocarriers to promote in-vivo delivery of oligonucleotides. Nanocarriers are known to enhance bioavailability and carrying capacity of siRNA payload; they also provide high interaction with target cells. Another advantage of nanoparticles as carriers is that according to their charge, they can interact with blood proteins to incite toxicity or even affect biodistribution.

Application Insights

Rising need for cure and prevention of diseases such as cancer, HIV, and other mutating viral diseases is anticipated to spur the usage rate of oligonucleotide products. Increasing number of clinical trials for oligonucleotide drugs exhibits that the field is expanding at a rapid pace. A case in point would be Ionis Pharmaceuticals; the company alone has four molecules in the pipeline and two approved molecules in the European Union and one in U.S.

Oncology has emerged as a notable segment within the market. Presence of a substantial pipeline for cancer therapies by companies and institutes such as Enzon Pharmaceuticals (Santaris Pharma), University of Texas, OncoGenex, Isarna Therapeutics, AstraZeneca (Ionis Pharmaceuticals), and INSYS Therapeutics Inc. is expected to drive this segment’s potential over the coming years.

Global antisense & RNAi therapeutics market

In addition to this, many companies have invested in R&D for nanocarriers to deliver oligonucleotides for cancer treatment, which is expected to contribute positively to cancer management. Nanocarriers can minimize the toxicity of these oligonucleotides. However, since nanoparticle systems are affected by inter- and intra-tumor heterogeneity, demand for efficient tumor targeting vectorization systems and multiple genes targeting oligonucleotide drugs is anticipated to increase in the coming years. This need for newer and safer delivery systems for management of cancer and other viral diseases is expected to propel market growth.

Regional Insights

North America is a key market, with U.S. reflecting a number of RNAi therapeutics currently in developmental pipelines. A number of biotechnology companies have made considerably high investments in RNAi therapeutic development. Big pharmaceutical developers have entered into collaboration agreements or licensing deals with a number of smaller firms in an attempt to capitalize on the expected revenue growth of this market. AstraZeneca’s agreement with Ionis Pharmaceuticals is one of the notable deals that reflect the trend of growing investments in antisense technology.

Since the discovery of RNAi, there have been numerous avenues of treatment development using RNAi. However, the market has picked up very little over the past decade due to complexities in delivery methodologies related to RNAi. Development of newer nanotechnology-oriented methods has re-infused the market with high growth potential over the forecast period.

A number of drug candidates under clinical trials have shown favorable results and are progressing through the development phases. Assuming that such trials continue to show positive results, it can be expected that the North America market will witness considerable growth in demand over the forecast period.

Antisense & RNAi Therapeutics Market Share Insights

GSK, Sanofi-Genzyme, Alnylam Pharmaceuticals, Marina Biosciences, and Benitec Biopharma are some of the prominent players operating in this market. These players are involved in development of RNAi therapies and platforms that offer medicines, diagnostics, and personalized treatment options for key diseases.

Alnylam Pharmaceuticals, Isis Pharmaceuticals/ Ionis Pharmaceuticals, Quark Pharmaceuticals, and Gene Signal are companies with a stronger pipeline portfolio. Acquisition of major participants as well as prominent emerging players has been observed in order to maintain market dominance.

Report Scope



Base year for estimation


Actual estimates/Historical data

2014 - 2017

Forecast period

2018 - 2025

Market representation

Revenue in USD Million and CAGR from 2018 to 2025

Regional scope

North America, Europe, Asia Pacific, Latin America, Middle East & Africa

Country scope

U.S., Canada, Germany, U.K., China, Japan, Brazil, South Africa

Report coverage

Revenue forecast, company share, competitive landscape, growth factors and trends

15% free customization scope (equivalent to 5-analyst working days)

If you need specific information, which is not currently within the scope of the report, we will provide it to you as a part of customization

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis on latest industry trends in each of the sub-segments from 2014 to 2025. For the purpose of this study, Grand View Research has segmented the global antisense & RNAi therapeutics market report on the basis technology, application, route of administration, and region:

  • Technology Outlook (Revenue, USD Million, 2014 - 2025)

    • RNA Interference

      • siRNA

      • miRNA

    • Antisense RNA

  • Application Outlook (Revenue, USD Million, 2014 - 2025)

    • Oncology

    • Cardiovascular

    • Respiratory Disorders

    • Renal Diseases

    • Neurodegenerative Disorders

    • Genetic Disorders

    • Infectious Diseases

    • Other

  • Route of Administration Outlook (Revenue, USD Million, 2014 - 2025)

    • Pulmonary Delivery

    • Intravenous Injections

    • Intradermal Injections

    • Intraperitoneal Injections

    • Topical Delivery

    • Other Delivery Methods

  • Regional Outlook (Revenue, USD Million, 2014 - 2025)

    • North America

      • U.S.

      • Canada

    • Europe

      • Germany

      • U.K.

    • Asia Pacific

      • Japan

      • China

    • Latin America

      • Brazil

    • Middle East & Africa

      • South Africa

Key questions answered by the report
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