RNA Targeting Small Molecules Therapeutics Global Market Report 2024

The rna targeting small molecules therapeutics market size has grown exponentially in recent years. It will grow from $1.06 billion in 2023 to $1.45 billion in 2024 at a compound annual growth rate (CAGR) of 37.0%. The increase observed in the historical period can be attributed to factors such as advancements in drug discovery and development, the rising prevalence of RNA-related diseases, financial support from government and private funding sources, the growing interest of the pharmaceutical industry in RNA therapeutics, and success stories associated with RNA therapeutics.

The rna targeting small molecules therapeutics market size is expected to see exponential growth in the next few years. It will grow to $5.09 billion in 2028 at a compound annual growth rate (CAGR) of 36.9%. The anticipated growth in the forecast period can be attributed to factors such as the increasing focus on precision medicine and personalized therapies, a rise in RNA biomarker identification, an expanding spectrum of RNA targets, growing funding and investments in RNA therapeutics, and their application in rare diseases. Notable trends expected during the forecast period include advancements in screening technologies, the dominance of antisense oligonucleotides, the development of small molecule RNA binders, the use of RNA therapeutics in neurological disorders, and the application of gene editing with CRISPR-Cas9 technology.

The anticipated rise in cancer cases is set to drive the growth of the RNA-targeting small molecule therapeutics market. Cancer, a diverse set of diseases characterized by uncontrolled cell proliferation, necessitates treatment with RNA-targeting small molecules designed to selectively target RNA molecules associated with cancer. As per research from the American Cancer Society in January 2022, the US is expected to witness 1.9 million new cancer cases and 609,360 cancer-related deaths in 2022. This alarming statistic, averaging 1,670 fatalities daily, underscores the imperative need for advancements in RNA-targeting small molecule therapeutics to combat the increasing number of cancer cases.

The surge in genetic disorders is poised to amplify the growth of the RNA-targeting small molecule therapeutics market. Genetic disorders, arising from abnormalities or mutations in an individual's genetic material, can be effectively addressed by RNA-targeting small molecules. This innovative field holds great potential for treating genetic disorders, particularly those involving toxic expansions of repeated RNA sequences. A case in point is sickle cell disease, which, according to a Lancet report in June 2023, witnessed a 41.4% increase globally, reaching 7.74 million in 2021. The rising incidence of genetic disorders, exemplified by this data, fuels the growth of the RNA-targeting small molecule therapeutics market.

The global rise in infectious diseases is anticipated to contribute to the growth of the RNA-targeting small molecule therapeutics market. These diseases, caused by pathogenic microorganisms, are being combated with RNA-targeting small molecules as a promising approach. By binding to specific structures within RNA molecules, these small molecules disrupt RNA function, hindering pathogen replication or virulence. For instance, considering the data from the Office for National Statistics, the increase in COVID-19 infections in England underscores the urgent need for RNA-targeting small molecule therapeutics to address the rising global infectious diseases.

Strategic partnerships have emerged as a significant trend in the RNA-targeting small molecule therapeutics market. Major companies within the market are forming partnerships, forging collaborative efforts to develop innovative products. In June 2022, Otsuka Pharmaceutical Co. Ltd. collaborated with xFOREST Therapeutics Co. Ltd., pooling resources and leveraging xFOREST's Forest technology for systemic small-molecule drug discovery research targeting RNA structures. Similarly, in March 2021, Evotec SE joined forces with Takeda Pharmaceutical Company Limited to undertake collaborative efforts in discovering and developing RNA-targeting small molecules.

In January 2021, Ribometrix, Inc., collaborated with Genentech Inc. to advance RNA-targeted therapeutic small molecules. This partnership between the US-based drug discovery company and Genentech aims to develop small-molecule drugs that modify RNA function by focusing on three-dimensional RNA structures. Genentech Inc. is a US-based biotechnology research company.

Major companies operating in the rna targeting small molecules therapeutics market report are F. Hoffmann-La Roche Ltd., Bayer AG, Sanofi S.A., AstraZeneca PLC, Eli Lilly and Company, Amgen Inc., Janssen Pharmaceuticals, Celgene Corporation, Vertex Pharmaceuticals, Servier laboratories, Genentech Inc., PTC Therapeutics Inc., Arrakis Therapeutics, Anima Biotech Inc., Skyhawk Therapeutics Inc., Remix Therapeutics, Abivax SA, 858 Therapeutics, Eloxx Pharmaceuticals Inc., STORM Therapeutics Ltd., Accent Therapeutics Inc., eFFECTOR Therapeutics Inc., EPICS Therapeutics, Expansion Therapeutics Inc., Inspirna Inc., MiNA Therapeutics Ltd., Ribometrix Inc., AC Immune SA, Redona Therapeutics.

North America was the largest region in the RNA targeting small molecules therapeutics market in 2023. The regions covered in the rna targeting small molecules therapeutics market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the rna targeting small molecules therapeutics market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The RNA-targeting small molecule therapeutics market consists of sales of nusinersen, inotersen, givosiran, and patisiran. Values in this market are ‘factory gate’ values, that is, the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.


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