The RNA targeting small molecule therapies market is projected to be worth around USD 8.3 billion in 2030

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In the recent years, RNA’s potential as a target molecule for small molecules has been widely recognized; as a result, a number of industry players have developed their proprietary technologies for direct and indirect RNA targeting and are actively engaged in the discovery and developmen

Roots Analysis has announced the addition of “RNA Targeting Small Molecules Therapeutics Market, 2021-2030” report to its list of offerings.


RNAs represent a one-of-a-kind class of molecules that can interact with three different types of biological macromolecules, namely DNA, RNA and proteins which further enables the development of therapies capable of targeting previously deemed undruggable transcripts and genes. In the past few years, a number of novel techniques have been developed to expand the druggability of RNA molecules. As a result, numerous small molecules, capable of acting on current and novel RNA targets are also being investigated in clinical and preclinical studies for the treatment of various indications, such as genetic diseases, infectious diseases and different types of cancers.


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Key Market Insights


100+ RNA targeting small molecule therapeutics have been / are being developed for the treatment of different types of disease indications

Close to 70% of the aforementioned therapeutic candidates are under preclinical evaluation. Further, two therapies, namely Translarna™ and Risdiplam, have been approved for the treatment of Duchenne Muscular Dystrophy and spinal muscular atrophy respectively.


More than 45% of the therapies under clinical evaluation are currently in phase II developmental stage

Around 50% of therapies under clinical evaluation are being developed to target oncological disorders; of which 53% of candidates are being evaluated under phase I clinical stage of development


Close to 60% of the therapies under preclinical evaluation are currently in discovery phase of development

Around 44% of the therapies under preclinical evaluation are being developed to target neurological disorders; of which close to 50% of drug candidates underwent RNA splicing modification.


More than 55% of the players evaluating RNA Targeting small molecule therapies are small companies

North America has emerged as a key hub, featuring the presence of 63% developers. The developer landscape is further dominated by players that have been established post-2012, representing around 68% of the total number of stakeholders


Partnership activity in this field has increased at a CAGR of 9.1%, between 2018 and 2020

More than 70% of the reported deals were RD agreements, majority of which were focused on drug candidates targeting neurological disorders. Further, majority of the instances captured in the report were inked with players based in North America (65%).


Close to USD 500 million was raised by companies involved in the development of drug candidates targeting oncological disorders

Majority of the companies (52%) engaged in this domain primarily received funding through venture capital rounds. Further, around 90% of the funding instances were reported by players headquartered in the US.


North America is anticipated to capture over ~70% of the global market share in 2030

In 2030, close to 40% of the market revenues are expected to be generated from sales of therapeutics that are intended to target oncological disorders. Further, therapies designed for oral route of administration are expected to occupy a larger share (77%) of the overall market in 2030.


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Key Questions Answered

  • Who are the key players engaged in the development of RNA targeting small molecule therapeutics?
  • What are the current investment trends in this field?
  • Which are the key therapeutic areas for which RNA targeting small molecule therapies are being developed?
  • What are the different technological approaches adopted by various players for the development of RNA targeting small molecule therapeutics?
  • What type of partnership models are most commonly adopted by stakeholders engaged in this domain?
  • What is the relative competitiveness of different RNA targeting small molecule therapeutics developers engaged in this market?
  • How is the current and future market opportunity likely to be distributed across key market segments?


The USD XX million (by 2030) financial opportunity within the RNA targeting small molecules therapeutics market has been analyzed across the following segments:


  • Drug
  • Translarna™
  • Risdiplam
  • Tomivosertib
  • ELX-02
  • H3B-6545
  • ABX464


  • Type of Indication
  • Aniridia
  • Breast Cancer
  • Castrate-resistant Prostate Cancer
  • Colorectal Cancer
  • Crohn Disease
  • Cystic Fibrosis
  • Duchenne Muscular Dystrophy
  • Dravet Syndrome
  • HIV Infections
  • Impaired Renal Function
  • Rheumatoid Arthritis
  • Spinal Muscular Atrophy
  • Ulcerative Colitis


  • Type of Therapeutic Area
  • Autoimmune Disorders
  • Genetic Disorders
  • Inflammatory Disorders
  • Infectious Disorders
  • Kidney Disorders
  • Neurological Disorders
  • Oncological Disorders
  • Ophthalmic Disorders
  • Rare Disorders
  • Respiratory Disorders


  • Type of Target Molecule
  • CDKL5
  • Cap Binding Complex
  • Dystrophin Protein
  • Estrogen Receptor Alpha
  • G542X
  • MNK1 / MNK2 PAX6 Gene
  • PAX6 Gene
  • SMN2


  • Type of Route of Administration
  • Oral
  • Subcutaneous


  • Key Geographical Regions
  • North America
  • Europe


The research includes profiles of key players (listed below); each profile features a brief overview of company, pipeline details, recent developments (including collaborations and expansions) and an informed future outlook.


  • Abivax
  • AC immune
  • Arrakis Therapeutics
  • eFFECTOR Therapeutics
  • Eloxx Pharmaceuticals
  • H3 Biomedicine
  • PTC Therapeutics
  • Ribometrix
  • Skyhawk Therapeutics
  • STORM Therapeutics



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