RNA Targeting Small Molecules Therapeutics Global Market Opportunities and Strategies to 2033

This report describes and explains the RNA targeting small molecule therapeutics market and covers 2018-2023, termed the historic period and 2023-2028, 2033F termed the forecast period. The report evaluates the market across each region and for the major economies within each region.

The global RNA targeting small molecule therapeutics market reached a value of nearly $4.8 billion in 2023, having grown at a compound annual growth rate (CAGR) of 28.8% since 2018. The market is expected to grow from $4.8 billion in 2023 to $17.3 billion in 2028 at a rate of 29.4%. The market is then expected to grow at a CAGR of 28.3% from 2028 and reach $60.2 billion in 2033.

Growth in the historic period resulted from the increasing incidence of neurological disorders, growing interest in personalized medicine and government initiatives for research and development in healthcare. Factors that negatively affected growth in the historic period were complexity of RNA-based drug discovery.

Going forward, increasing government funding for RNA research, rising prevalence of cancer and increasing incidence of genetic disorders will drive the growth. Factor that could hinder the growth of the RNA targeting small molecule therapeutics market in the future include high cost of drug development.

The RNA targeting small molecule therapeutics market is segmented by product into antisense oligonucleotides (ASOs) and small interfering RNA (siRNA). The antisense oligonucleotides (ASOs) market was the largest segment of the RNA targeting small molecule therapeutics market segmented by type, accounting for 68.8% or $3.3 billion of the total in 2023. Going forward, the siRNA is expected to be the fastest growing segment in the RNA targeting small molecule therapeutics market segmented by type, at a CAGR of 30.6% during 2023-2028.

The RNA targeting small molecule therapeutics market is segmented by therapeutic indication into musculoskeletal disorder, neurodegenerative diseases, cardiovascular disease, genito-urinary and therapeutic indications. The musculoskeletal disorder market was the largest segment of the RNA targeting small molecule therapeutics market segmented by therapeutic indication, accounting for 61.7% or $2.9 billion of the total in 2023. Going forward, the cardiovascular disease segment is expected to be the fastest growing segment in the RNA targeting small molecule therapeutics market segmented by therapeutic indication, at a CAGR of 32.6% during 2023-2028.

The RNA targeting small molecule therapeutics market is segmented by end users into hospitals, research laboratories, pharmaceutical and biotechnology companies and other end-users. The pharmaceutical and biotechnology companies market was the largest segment of the RNA targeting small molecule therapeutics market segmented by end-user, accounting for 55.1% or $2.6 billion of the total in 2023. Going forward, the research laboratories segment is expected to be the fastest growing segment in the RNA targeting small molecule therapeutics market segmented by end-user, at a CAGR of 30.3% during 2023-2028.

North America was the largest region in the RNA targeting small molecule therapeutics market, accounting for 52.8% or $2.5 billion of the total in 2023. It was followed by Western Europe, Asia Pacific and then the other regions. Going forward, the fastest-growing regions in the RNA targeting small molecule therapeutics market will be South America and Eastern Europe where growth will be at CAGRs of 35% and 31.6% respectively. These will be followed by Asia Pacific and Western Europe where the markets are expected to grow at CAGRs of 30.6% and 28.9% respectively.

The global RNA targeting small molecule therapeutics market is highly concentrated, with a few large players operating in the market. The top ten competitors in the market made up to 98.9% of the total market in 2022. The market concentration can be attributed to the presence of large number of players in different geographies. Prominent players are acquiring the products and entering into partnerships with the other companies to consolidate their market positions across the globe, while others are distributing products. Ionis Pharmaceuticals was the largest competitor with a 43.8% share of the market, followed by Alnylam Pharmaceuticals with 21.8%, Sarepta Therapeutics with 20.6%, Jazz Pharmaceuticals with 4.7%, Novartis with 2.7%, NS Pharma with 2.5%, Arrakis Therapeutics with 1.2%, PTC Therapeutics with 0.6%, Anima Biotech Inc. with 0.5% and Ribometrix with 0.5%.

The top opportunities in the RNA targeting small molecule therapeutics market segmented by type will arise in the ASO segment, which will gain $8.4 billion of global annual sales by 2028. The top opportunities in the RNA targeting small molecule therapeutics market segmented by therapeutic indication will arise in the musculoskeletal disorder segment, which will gain $8.4 billion of global annual sales by 2028. The top opportunities in the RNA targeting small molecule therapeutics market segmented by end-user will arise in the pharmaceutical and biotechnology companies segment, which will gain $7.1 billion of global annual sales by 2028. The RNA targeting small molecule therapeutics market size will gain the most in the USA at $4.6 billion.

Market-trend-based strategies for the RNA targeting small molecule therapeutics market include strategic collaboration fueling RNA targeting small molecules therapeutics, key players embrace startup solutions in RNA targeting small molecules therapeutics, use of artificial intelligence (AI) to enhance efficiency of RNA targeting small molecules during drug discovery, partnerships driving innovation in RNA targeting small molecules therapeutics, focus on advancement in RNA degrader therapeutics targeting for various medical domains, increased focus on mRNA small molecule drug discovery and development of RNA modulatory drugs for treating neurological diseases.

Player-adopted strategies in the RNA targeting small molecule therapeutics market include expanding capabilities in to boost the commercial potential of offerings, expanding capabilities in RNA interference (RNAi) therapeutics to deliver transformative medicines in both rare and common diseases, expanding capabilities in gene therapy manufacturing and strategic partnerships to advance its pipeline efficiently and effectively and enhancing operational capabilities through strategic mergers and acquisitions.

To take advantage of the opportunities, the analyst recommends RNA targeting small molecules therapeutics companies to focus on AI integration for enhanced drug discovery, focus on advancing RNA degrader therapeutics, focus on MRNA small molecule drug discovery, focus on SIRNA market segment, focus on musculoskeletal disorder and cardiovascular disease segments, expand in emerging markets, continue to focus on developed markets, focus on strategic collaborations for accelerated innovation, provide competitively priced offerings, continue to use B2B promotions, focus on pharmaceutical and biotechnology companies and research laboratories and focus on cancer patients.